Encaleret sulfate

- $243.7 million in total second quarter revenues, primarily comprised of $222.4 million of U.S. Attruby® net product revenue, with growth led by the treatment-naïve segment as physicians increasingly start and keep patients on Attruby - Attruby is the first ATTR-CM therapy associated with direct kidney protection, with post-hoc analyses published in Circulation: Heart Failure showing a pro with ACE inhibitors, ARBs, and SGLT2s including an early, reversible eGFR dip, an improved chronic eGFR slope relative to placebo, and a 13.7% reduction in urinary albumin-to-creatinine ratio through Month 30; the magnitude of the acute eGFR dip was positively associated with greater early cardiovascular benefit; BridgeBio will explore the potential for Attruby to treat other orphan kidney indications - Real-world evidence continues to differentiate Attruby from tafamidis, with an independent propensity score-matched analysis of 286 patient pairs from the TriNetX network published in JSCAI associating acoramidis with a 37% reduction in composite cardiovascular events (p=0.002) and a 34% reduction in hospitalizations (p=0.002) at six months; further independent RWE using electronic health records are expected, and we are confident Attruby will consistently demonstrate clinical superiority over tafamidis to the benefit of patients and healthcare delivery systems for which heart failure remains a top concern - All three planned NDAs are now submitted to the FDA: BBP-418 for LGMD2I/R9 was accepted with Priority Review (PDUFA November 27, 2026); encaleret for ADH1 was accepted with Priority Review (PDUFA May 8, 2027), with no advisory committee planned for either; oral infigratinib for achondroplasia has been submitted, with U.S. launch expected mid-2027 ezstandalone.cmd.push(function () { ezstandalone.showAds(131); }); - Diagnosis and awareness continue to accelerate ahead of the launches: in ADH, more than 2,200 unique patients are now identified under the dedicated ICD-10 code, at approximately 70 new diagnoses per month; in LGMD2I/R9, BridgeBio is investing in awareness and multidisciplinary care at MDA Care Center Network sites, where we expect 85% of target physicians to be familiar with the BBP-418 pro data by launch - The oral encaleret and oral infigratinib franchises continue to expand beyond their first indications: RECLAIM-HP in chronic hypoparathyroidism has begun screening patients with topline data anticipated in late 2027 or early 2028, CALIBRATE-PEDS in pediatric ADH1 has completed enrollment in its first cohort, and a Phase 2 update in hypochondroplasia is expected in the second half of 2026 - $720.2 million in cash, cash equivalents, and marketable securities as of June 30, 2026, which does not include the $1 billion preferred equity financing that closed on July 1, 2026 - BridgeBio will host a Commercial Day in New York City on October 8, 2026, to discuss commercial readiness and launch strategy across its three upcoming launches PALO ALTO, Calif., Aug. 10, 2026 (GLOBE NEWSWIRE) -- BridgeBio Pharma, Inc. (Nasdaq: BBIO) (“BridgeBio” or the “Company”), a commercial-stage, multi-product biopharmaceutical company focused on developing medicines for genetic conditions, announced today its financial results for the second quarter ended June 30, 2026, and provided an update on Attruby’s commercial progress. Pipeline Overview: Program Status Next expected milestone Acoramidis for ATTR-CM Approved in U.S., E.U., Japan, Switzerland, Brazil, and U.K. New data to be shared at ESC 2026 BBP-418 for LGMD2I/R9 PDUFA date set for November 27, 2026 with Priority Review Launch upon FDA approval Encaleret for ADH1 PDUFA date set for May 8, 2027 with Priority Review; MAA submitted to EMA Launch upon FDA approval Oral infigratinib for achondroplasia NDA submitted to FDA FDA sets PDUFA date Oral Encaleret for chronic hypoparathyroidism First investigational sites activated for RECLAIM-HP, Phase 3 study First participant dosed in Q3 2026 Oral infigratinib for hypochondroplasia ACCEL 2/3 enrollment ongoing Phase 2 clinical trial update in 2H 2026 Depleter for ATTR-CM Development candidate nomination Submit IND to the FDA in 2027 “I'm excited by the growing body of evidence continuing to demonstrate Attruby is the drug of choice for all ATTR-CM patients, and particularly for those who are treatment-naïve, including the first-ever demonstration of early, sustained kidney-protective effects in ATTR-CM alongside the cardiac benefit we've established. Furthermore, this was the quarter all three of our pipeline programs, BBP-418, encaleret, and infigratinib, moved from data into active regulatory review, with our first PDUFA date now set for November 27, 2026, which is a level of strategic execution and discipline I'm proud of. Finally, with the $1 billion preferred equity financing we completed, we have a balance sheet sized to run all three launches at full strength, without diverting resources from the development engine that produced them,” said Neil Kumar, Ph.D., Co-Founder and CEO of BridgeBio. Commercial Updates: The second quarter total revenues, net totaled $243.7 million, comprised of $222.4 million of U.S. Attruby net product revenue, $15.4 million from royalty revenue, and $5.8 million in license and services revenue. “We continue to see strong growth this quarter for Attruby with our first-line share climbing again,” said Matt Outten, Chief Commercial Officer of BridgeBio. “What comes next will continue to shape BridgeBio’s next chapter as we prepare for three potential approvals in three different diseases, all with best-in-class potential, each backed by the same commercial engine that made Attruby a success. We look forward to continuing to deliver for patients and addressing the gaps within the treatment paradigm for rare disease.” Pipeline Updates: Attruby (acoramidis) – First and only near-complete (≥90%) transthyretin (TTR) stabilizer for treatment of transthyretin amyloid cardiomyopathy (ATTR-CM): New post-hoc analyses published in Circulation: Heart Failure showed acoramidis was associated with a rapid, reversible estimated glomerular filtration rate (eGFR) dip alongside a placebo-corrected 15.5% reduction in urinary albumin-to-creatinine ratio (UACR) by Day 28, followed by a sustained improvement in chronic eGFR slope (+2.47 mL/min/1.73m²/year) and a 13.7% UACR reduction through Month 30. This pro that of direct-acting kidney medicines such as ACE inhibitors, ARBs, and SGLT2 inhibitors, and has not previously been observed with any approved ATTR-CM therapy. Participants with larger eGFR dips had a 58% lower risk of death or cardiovascular hospitalization in year one, suggesting the kidney effect may contribute to acoramidis' early cardiovascular benefit. New data from ATTRibute-CM presented in two late-breaking oral presentations at Heart Failure 2026 further demonstrated acoramidis’ differentiated clinical profile. The first showed a reduction in the risk of outpatient worsening heart failure by 41% versus placebo with separation of curves seen within 30 days and sustained through Month 30. The second showed a significant reduction in serum transthyretin variability, which is associated with lower mortality. Real-world evidence continues to demonstrate that Attruby is differentiated from other therapies in the speed and strength of benefit. An independent propensity score-matched analysis of 286 patient pairs from the TriNetX network, presented at SCAI 2026 Scientific Sessions and published in JSCAI , associated acoramidis with a 37% reduction in composite cardiovascular events (HR 0.63; p=0.002) and a 34% reduction in hospitalizations (HR 0.67; p=0.002) at six months, with effects deepening at nine months and significant reductions across heart failure exacerbation, arrhythmia, and acute kidney injury. A separate propensity score-weighted analysis observed a 43% reduction in outpatient diuretic intensification (HR 0.57; p=0.021) versus tafamidis. A third independent study conducted in EPIC COSMOS and to be published later in 2026 validated the comparative effectiveness of acoramidis over tafamidis. These three real-world datasets converge on consistent, statistically significant benefit in contemporary patients on modern background therapy, reinforcing that Attruby's stabilization advantage is showing up in outcomes that matter to physicians, patients, and payers. BridgeBio initiated ASCEND-ATTR, a Phase 4 study using cardiac MRI and echocardiography to characterize the long-term effects of acoramidis on disease reversal as measured by cardiac structure, function, and amyloid burden over 36 months. This builds on the evidence of regression observed in ATTRibute-CM and the open-label extension. Additional data will be shared in two oral presentations and six moderated posters at the European Society of Cardiology (ESC) Congress 2026. BBP-418 – Glycosylation substrate for limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9): BridgeBio believes BBP-418 is positioned to become the first approved therapy for individuals living with LGMD2I/R9, addressing a significant unmet need in this disease and potentially representing the first approval of a therapy for any form of LGMD. On May 27, 2026, the FDA accepted the Company's New Drug Application (NDA) for BBP-418 and granted Priority Review, assigning a Prescription Drug User Fee Act (PDUFA) target action date of November 27, 2026. No advisory committee meeting is currently planned. FORTIFY, the Phase 3 clinical trial of BBP-418, successfully met all pre-specified primary and secondary endpoints of its 12-month interim analysis, supporting its potential as a disease-modifying therapy. The topline results can be found here . Additional positive results demonstrating the rapid and consistent treatment effect and favorable safety pro BBP-418 were presented in March at the 2026 MDA Clinical and Scientific Conference in a late-breaking oral presentation. 1 At the 19th International Congress on Neuromuscular Diseases in July 2026, BridgeBio presented interim FORTIFY data showing favorable patient-reported outcomes for BBP-418-treated individuals compared to placebo, demonstrating that the improvements observed on functional outcomes and biomarkers translate to how patients feel and function. BridgeBio invested $100,000 to strengthen multidisciplinary LGMD care through a Muscular Dystrophy Association (MDA) Care Advance Grant supporting initiatives at Stanford Health Care and the University of Minnesota and engaged the LGMD2I/R9 community at the 2026 European LGMD2I/R9 Conference and the 2026 Iowa Wellstone Dystroglycanopathy Patient & Family Conference. BridgeBio's neuromuscular U.S. field teams are hired, trained, and deployed across medical, commercial, and market access, engaging in scientific exchange, disease state education, and account profiling as appropriate in the pre-approval setting. Promotional activity will commence only upon FDA approval, consistent with regulatory requirements. Based on the FORTIFY interim analysis results, BridgeBio is also engaging regulatory agencies to identify an expedited path to approval for BBP-418 in Europe. The Company intends to initiate clinical studies of BBP-418 in LGMD2I/R9 for individuals less than 12 years of age in the first half of 2027, and in LGMD2M/R13 and LGMD2U/R20 in the near future. Encaleret – Calcium-sensing receptor (CaSR) antagonist for autosomal dominant hypocalcemia type 1 (ADH1) and chronic hypoparathyroidism: BridgeBio believes encaleret is positioned to become the first approved therapy specifically indicated for individuals living with ADH1, in both the U.S. and the EU. The FDA re-considered its review designation of the Company's NDA filing for encaleret in ADH1 and has granted Priority Review, with a PDUFA target action date of May 8, 2027. No advisory committee meeting is currently planned. BridgeBio submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for the use of encaleret in ADH1. Diagnosis of ADH1 in the U.S. continues to accelerate, with more than 2,200 unique patients identified under the dedicated ICD-10 code for ADH (E20.810) from its introduction in October 2023 through June 2026, a rate of approximately 70 new diagnoses per month. CALIBRATE-PEDS, the registrational Phase 2/3 study of encaleret in pediatric ADH1, completed enrollment in the first of four cohorts (adolescents 12 to 17 years of age). RECLAIM-HP, the Phase 3 study of encaleret in chronic hypoparathyroidism, has activated its first investigational sites and screening has initiated. Chronic hypoparathyroidism affects approximately 200,000 patients in the U.S. and EU, and represents a substantial expansion of the encaleret opportunity beyond ADH1. Oral infigratinib – FGFR3 inhibitor for achondroplasia and hypochondroplasia: BridgeBio believes oral infigratinib is positioned to become the first approved oral therapy and a potential best-in-class option for children living with achondroplasia and hypochondroplasia. The Company submitted an NDA to the FDA for oral infigratinib in achondroplasia and is on track to submit an MAA to the EMA in the fourth quarter of 2026. BridgeBio anticipates a U.S. launch in mid-2027 and an EU approval in the second half of 2027. Oral infigratinib has received Breakthrough Therapy, Fast Track, and Rare Pediatric Disease designations from the FDA, and Orphan Drug designation from both the FDA and the EMA. Results from PROPEL 3, the Phase 3 trial of oral infigratinib in achondroplasia, were published in the New England Journal of Medicine 2 and simultaneously presented at the International Congress of Children's Bone Health 2026. Oral infigratinib is the only achondroplasia program with Phase 3 results published in the New England Journal of Medicine. In these results 3 , oral infigratinib significantly improved arm span Z-score versus placebo (LS mean +0.37 SD; p<0.0001) at 52 weeks, the first statistically significant placebo-controlled arm span improvement reported in an achondroplasia trial. PROPEL 3 achieved its pre-specified primary efficacy endpoint of change from baseline in absolute height velocity at Week 52 (p<0.0001), and oral infigratinib showed the first statistically significant improvement in body proportionality against placebo in achondroplasia in children 3 to younger than 8 years old in a pre-specified exploratory analysis. Oral infigratinib remains the only therapy to demonstrate efficacy measures beyond annualized height velocity and height Z-score in a placebo-controlled setting at 52 weeks. The topline results can be found here . U.S. commercial and medical affairs leadership are in place, with the field medical team fully onboarded and regional sales directors building field teams aligned to the diagnosed and treatable population living with achondroplasia. The Company has initiated development of the achondroplasia program from birth to under 3 years of age and is actively enrolling participants. The Company is enrolling participants in the observational run-in study for the Phase 3 trial in hypochondroplasia. An update of the Phase 2 program in hypochondroplasia is expected in the second half of 2026. _______________ 1 2 3 Corporate Updates: On July 1, 2026, BridgeBio closed $1 billion in preferred equity led by Sixth Street with participation from HealthCare Royalty, an affiliate of KKR. The financing enables BridgeBio to fund three potential upcoming launches while continuing to invest in indication expansion across the pipeline. Financial Updates: Cash, Cash Equivalents and Marketable Securities Cash, cash equivalents and marketable securities totaled $720.2 million and $587.5 million as of June 30, 2026 and December 31, 2025, respectively. Total Revenues, Net Three Months Ended June 30, Six Months Ended June 30, 2026 2025 2026 2025 (in thousands) Net product revenue $ 222,440 $ 71,501 $ 403,036 $ 108,240 License and services revenue 5,804 37,440 10,223 117,130 Royalty revenue 15,432 1,624 24,932 1,828 Total revenues, net $ 243,676 $ 110,565 $ 438,191 $ 227,198 Total revenues, net for the three months ended June 30, 2026 were $243.7 million compared to $110.6 million for the same period in 2025. The $133.1 million increase was primarily driven by a $150.9 million increase in net product revenue from Attruby, and a $13.8 million increase in royalty revenue primarily earned from net product sales of BEYONTTRA in the EU and Japan. These increases were partially offset by a decrease in license and services revenue primarily due to recognition of $30.0 million of regulatory milestone-related revenue during the three months ended June 30, 2025. Total revenues, net for the six months ended June 30, 2026 were $438.2 million compared to $227.2 million for the same period in 2025. The $211.0 million increase was primarily driven by a $294.8 million increase in net product revenue from Attruby, and a $23.1 million increase in royalty revenue primarily earned from net product sales of BEYONTTRA in the EU and Japan. These increases were partially offset by a decrease in license and services revenue primarily due to recognition of $105.0 million of regulatory milestone-related revenues during the six months ended June 30, 2025. Total Operating Costs and Expenses Three Months Ended June 30, Six Months Ended June 30, 2026 2025 2026 2025 (in thousands) Total cost of revenues $ 15,046 $ 3,653 $ 24,985 $ 6,292 Research and development 149,448 111,231 276,084 222,662 Selling, general and administrative 186,261 129,154 350,157 235,519 Restructuring, impairment, and related charges — 805 — 1,375 Total operating costs and expenses $ 350,755 $ 244,843 $ 651,226 $ 465,848 Total operating costs and expenses for the three months ended June 30, 2026 were $350.8 million, compared to $244.8 million for the same period in 2025. The $106.0 million increase was primarily driven by a $57.1 million increase in selling, general and administrative (SG&A) expenses, reflecting continued investment in both the ongoing commercialization of Attruby and the pre-commercial activities for BridgeBio's Phase 3 product candidates, a $38.2 million increase in research and development (R&D) expenses to support the development of late-stage product candidates, and an $11.4 million increase in total cost of revenues primarily due to higher sales volume of Attruby. Total operating costs and expenses for the six months ended June 30, 2026 were $651.2 million, compared to $465.8 million for the same period in 2025. The $185.4 million increase was primarily driven by a $114.6 million increase in SG&A expenses, reflecting continued investment in both the ongoing commercialization of Attruby and the pre-commercial activities for BridgeBio's Phase 3 product candidates, an $18.7 million increase in total cost of revenues primarily due to higher sales volume of Attruby, and a $53.4 million increase in R&D expenses to support headcount growth and the development of late-stage product candidates. Stock-based compensation expenses included in operating costs and expenses for the three months ended June 30, 2026 were $44.5 million, of which $28.4 million, $15.4 million, and $0.7 million were included in SG&A expenses, R&D expenses, and cost of goods sold, respectively. Stock-based compensation expenses included in operating costs and expenses for the same period in 2025 were $37.3 million, of which $23.2 million, $14.0 million, and $0.1 million were included in SG&A expenses, R&D expenses, and cost of goods sold, respectively. Stock-based compensation expenses included in operating costs and expenses for the six months ended June 30, 2026 were $77.9 million, of which $48.6 million, $27.6 million, and $1.7 million were included in SG&A expenses, R&D expenses, and cost of goods sold, respectively. Stock-based compensation expenses included in operating costs and expenses for the same period in 2025 were $66.7 million, of which $41.2 million, $25.3 million, and $0.2 million were included in SG&A expenses, R&D expenses, and cost of goods sold, respectively. Total Other Expense, Net Total other expense, net for the three and six months ended June 30, 2026, was $(48.8) million and $(109.4) million, respectively, compared to $(47.4) million and $(112.6) million, respectively, for the same periods in 2025. The increase in total other expense, net of $1.4 million for the three months ended June 30, 2026, compared to the same period in 2025 was primarily driven by a $15.3 million increase in noncash interest expense related to deferred royalty obligations, and was partially offset by a $13.8 million decrease in net loss from equity method investments. The decrease in total other expense, net of $3.2 million for the six months ended June 30, 2026, compared to the same period in 2025 was primarily driven by a $21.2 million decrease in loss on extinguishment of debt recognized in 2025, and an $11.0 million decrease in net loss from equity method investments. These decreases were partially offset by a $31.2 million increase in noncash interest expense related to deferred royalty obligations. Net Loss Attributable to Common Stockholders of BridgeBio and Net Loss per Share For the three and six months ended June 30, 2026, the Company recorded a net loss attributable to common stockholders of BridgeBio of $152.2 million and $316.3 million, respectively, compared to $181.9 million and $349.3 million, respectively, for the same periods in 2025. For the three and six months ended June 30, 2026, the Company reported a net loss per share of $0.78 and $1.62, respectively, compared to $0.95 and $1.84, respectively, for the same periods in 2025. BRIDGEBIO PHARMA, INC. Condensed Consolidated Statements of Operations (in thousands, except share and per share amounts) Three Months Ended June 30, Six Months Ended June 30, 2026 2025 2026 2025 (Unaudited) (Unaudited) Revenues: Net product revenue $ 222,440 $ 71,501 $ 403,036 $ 108,240 License and services revenue 5,804 37,440 10,223 117,130 Royalty revenue 15,432 1,624 24,932 1,828 Total revenues, net 243,676 110,565 438,191 227,198 Operating costs and expenses: Cost of revenues: Cost of goods sold 10,528 2,848 18,260 4,882 Cost of license, services, and royalty revenue 4,518 805 6,725 1,410 Total cost of revenues 15,046 3,653 24,985 6,292 Research and development 149,448 111,231 276,084 222,662 Selling, general and administrative 186,261 129,154 350,157 235,519 Restructuring, impairment, and related charges — 805 — 1,375 Total operating costs and expenses 350,755 244,843 651,226 465,848 Loss from operations (107,079 ) (134,278 ) (213,035 ) (238,650 ) Other income (expense), net: Interest income 6,659 3,898 12,905 9,283 Interest expense (13,278 ) (11,607 ) (26,220 ) (29,728 ) Noncash interest expense on deferred royalty obligations (1) (41,345 ) (26,030 ) (81,218 ) (50,050 ) Loss on extinguishment of debt — — — (21,155 ) Net loss from equity method investments (6,435 ) (20,189 ) (24,718 ) (35,745 ) Other income, net 5,587 6,548 9,840 14,779 Total other expense, net (48,812 ) (47,380 ) (109,411 ) (112,616 ) Loss before income taxes (155,891 ) (181,658 ) (322,446 ) (351,266 ) Provision for income taxes — 2,100 — 2,100 Net loss (155,891 ) (183,758 ) (322,446 ) (353,366 ) Net loss attributable to redeemable convertible noncontrolling interests and noncontrolling interests 3,675 1,855 6,187 4,041 Net loss attributable to common stockholders of BridgeBio $ (152,216 ) $ (181,903 ) $ (316,259 ) $ (349,325 ) Net loss per share attributable to common stockholders of BridgeBio, basic and diluted $ (0.78 ) $ (0.95 ) $ (1.62 ) $ (1.84 ) Weighted-average shares used in computing net loss per share attributable to common stockholders of BridgeBio, basic and diluted 195,785,884 190,517,215 195,290,642 190,332,261 (1) Including related party amounts of $(5,575) and $(10,936), respectively, for the three and six months ended June 30, 2026. Three Months Ended June 30, Six Months Ended June 30, Stock-based Compensation 2026 2025 2026 2025 (Unaudited) (Unaudited) Cost of goods sold $ 685 $ 126 $ 1,655 $ 217 Research and development 15,439 14,000 27,583 25,255 Selling, general and administrative 28,398 23,213 48,647 41,211 Restructuring, impairment and related charges — — — 46 Total stock-based compensation $ 44,522 $ 37,339 $ 77,885 $ 66,729 BRIDGEBIO PHARMA, INC. Condensed Consolidated Balance Sheets (In thousands) June 30, 2026 December 31, 2025 (Unaudited) (1) Assets Cash, cash equivalents and marketable securities $ 720,160 $ 587,482 Accounts receivable, net 254,484 139,444 Inventories 52,842 26,753 Prepaid expenses and other current assets 65,652 44,070 Equity method investments 55,095 79,972 Property and equipment, net 5,226 5,366 Operating lease right-of-use assets 17,079 8,149 Intangible assets, net 26,641 28,077 Other assets 20,120 16,712 Total assets $ 1,217,299 $ 936,025 Liabilities, Redeemable Convertible Noncontrolling Interests and Stockholders' Deficit Accounts payable $ 27,202 $ 36,228 Accrued and other current liabilities (2) 306,630 238,361 Operating lease liabilities 18,288 10,003 Deferred revenue 15,970 20,270 2027 Notes, net 547,955 547,015 2029 Notes, net 741,918 740,890 2031 Notes, net 565,528 564,565 2033 Notes, net 620,087 — Deferred royalty obligations, net (3) 879,374 855,030 Other long-term liabilities 240 244 Redeemable convertible noncontrolling interests (1,018 ) (570 ) Total BridgeBio stockholders' deficit (2,515,215 ) (2,086,610 ) Noncontrolling interests 10,340 10,599 Total liabilities, redeemable convertible noncontrolling interests and stockholders' deficit $ 1,217,299 $ 936,025 (1) The condensed consolidated balance sheet as of December 31, 2025 is derived from the audited consolidated financial statements as of that date. (2) Including related party amounts of $5,626 and $2,003 as of June 30, 2026 and December 31, 2025, respectively. (3) Including related party amounts of $206,208 and $204,650 as of June 30, 2026 and December 31, 2025, respectively. BRIDGEBIO PHARMA, INC. Condensed Consolidated Statements of Cash Flows (In thousands) Six Months Ended June 30, 2026 2025 (Unaudited) Operating activities: Net loss $ (322,446 ) $ (353,366 ) Adjustments to reconcile net loss to net cash used in operating activities: Stock-based compensation 77,314 63,123 Net loss from equity method investments 24,718 35,745 Noncash interest expense on deferred royalty obligations (1) 81,218 50,050 Change in fair value of the embedded derivative associated with the deferred royalty obligation (5,730 ) (5,499 ) Amortization of debt discount and issuance costs 3,745 3,056 Depreciation and amortization 2,239 2,601 Noncash lease expense 3,094 2,230 Loss on extinguishment of debt — 21,155 Dividend from investment in equity securities — (2,302 ) Other noncash adjustments, net 1,502 35 Changes in operating assets and liabilities: Accounts receivable, net (115,040 ) (72,146 ) Inventories (28,166 ) (16,582 ) Prepaid expenses and other current assets (20,227 ) (22,745 ) Other assets (3,283 ) (174 ) Accounts payable (9,026 ) 16,516 Accrued compensation and benefits (25,755 ) (15,637 ) Accrued research and development liabilities 23,032 (2,977 ) Operating lease liabilities (3,674 ) (3,117 ) Deferred revenue (4,300 ) (6,491 ) Other liabilities (2) 52,405 26,609 Net cash used in operating activities (268,380 ) (279,916 ) Investing activities: Purchases of marketable securities (63,937 ) (7,908 ) Maturities of marketable securities 39,600 — Payment for intangible assets — (6,095 ) Purchases of property and equipment (512 ) (594 ) Net cash used in investing activities (24,849 ) (14,597 ) Financing activities: Proceeds from issuance of 2033 Notes 632,500 — Issuance costs and discounts associated with 2033 Notes (13,227 ) — Proceeds from issuance of 2031 Notes — 575,000 Issuance costs and discounts associated with 2031 Notes — (12,034 ) Proceeds from royalty obligation under Royalty Purchase Agreement (as described in Note 9) — 300,000 Issuance costs associated with royalty obligation under Royalty Purchase Agreement — (2,012 ) Repayment of term loans — (459,000 ) Repayments of deferred royalty obligations (3) (33,082 ) (2,005 ) Repurchase of common stock (210,003 ) (48,276 ) Repurchase of RSU shares to satisfy tax withholding (8,341 ) (3,771 ) Proceeds from stock option exercises, net of repurchases 27,812 9,680 Proceeds from common stock issuances under ESPP 5,466 3,237 Transactions with noncontrolling interests — 1,550 Net cash provided by financing activities 401,125 362,369 Net increase in cash, cash equivalents, and restricted cash 107,896 67,856 Cash, cash equivalents, and restricted cash at beginning of period 572,140 683,244 Cash, cash equivalents, and restricted cash at end of period $ 680,036 $ 751,100 (1) Including a related party amount of $10,936 for the six months ended June 30, 2026. (2) Including a related party amount of $5,626 for the six months ended June 30, 2026. (3) Including a related party amount of $(5,784) for the six months ended June 30, 2026. Six Months Ended June 30, 2026 2025 (Unaudited) Supplemental Disclosure of Cash Flow Information: Cash paid for interest $ 20,316 $ 23,271 Supplemental Disclosures of Noncash Investing and Financing Information: Transfers to noncontrolling interests $ (5,464 ) $ (1,640 ) Recognized intangible asset recorded to “Accrued and other current liabilities” $ — $ 2,400 Deferred and unpaid issuance costs recorded to “Accrued and other current liabilities” $ 1,355 $ 998 Reconciliation of Cash, Cash Equivalents and Restricted Cash: Cash and cash equivalents $ 677,911 $ 748,953 Restricted cash — Included in “Prepaid expenses and other current assets” 549 449 Restricted cash — Included in “Other assets” 1,576 1,698 Total cash, cash equivalents and restricted cash at end of periods shown on the condensed consolidated statements of cash flows $ 680,036 $ 751,100 Webcast Information BridgeBio will host a conference call and webcast to discuss second quarter financial results today, August 10, 2026, at 4:30 pm ET. This event can be accessed at or by visiting the “Events & Presentations” page within the Investors section of the BridgeBio website at . A replay of the webcast will be available on the BridgeBio website for 30 days following the event. About Attruby ® (acoramidis) INDICATION Attruby is a transthyretin stabilizer indicated for the treatment of the cardiomyopathy of wild-type or variant transthyretin-mediated amyloidosis (ATTR-CM) in adults to reduce cardiovascular death and cardiovascular-related hospitalization. IMPORTANT SAFETY INFORMATION Adverse Reactions Diarrhea (11.6% vs 7.6%) and upper abdominal pain (5.5% vs 1.4%) were reported in patients treated with Attruby versus placebo, respectively. The majority of these adverse reactions were mild and resolved without drug discontinuation. Discontinuation rates due to adverse events were similar between patients treated with Attruby versus placebo (9.3% and 8.5%, respectively). About BridgeBio Pharma, Inc. BridgeBio exists to develop transformative medicines for genetic conditions. Millions of people worldwide living with genetic conditions lack treatment options, often because drug development for small patient populations can be commercially challenging. We aim to bridge the gap between advancements in genetic science and meaningful medicines for underserved patient populations. Our decentralized, hub-and-spoke model is designed for speed, precision, and scalability. Autonomous and empowered teams focus on individual conditions, while a central hub provides the clinical, regulatory, and commercial capabilities needed to bring innovation to market. For more information, visit bridgebio.com and follow us on LinkedIn , X , Facebook , Instagram , YouTube , and TikTok . BridgeBio Pharma, Inc. Forward-Looking Statements This press release contains forward-looking statements. Statements in this press release may include statements that are not historical facts and are considered forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended (the Securities Act), and Section 21E of the Securities Exchange Act of 1934, as amended (the Exchange Act), which are usually identified by the use of words such as “anticipates,” “believes,” “continues,” “estimates,” “expects,” “hopes,” “intends,” “may,” “plans,” “projects,” “remains,” “seeks,” “should,” “will,” and variations of such words or similar expressions. BridgeBio intends these forward-looking statements to be covered by the safe harbor provisions for forward-looking statements contained in Section 27A of the Securities Act and Section 21E of the Exchange Act. These forward-looking statements, including express and implied statements relating to the continued commercial success and market potential of Attruby/Beyonttra (acoramidis); the Company’s expectations regarding timing of regulatory submissions and target action dates, approvals and commercial launches in the U.S. and Europe, including for BBP-418 in LGMD2I/R9, encaleret in ADH1, and infigratinib in achondroplasia; the Company’s expectations regarding the timing and outcome of pre-commercial activities, including activities designed to support three potential launches; the timing of the Company’s clinical trials, milestones and expected updates for its various programs and pipeline; the safety and the potential benefits of the Company’s product and product candidates; the Company’s anticipated presentations of data; and the Company’s belief that its recent financing will enable the Company to fund its potential launches and expansion across its pipeline. Such statements reflect the Company’s current views about the Company’s plans, intentions, expectations and strategies, which are based on the information currently available to it and on assumptions the Company has made. Although the Company believes that its plans, intentions, expectations and strategies as reflected in or suggested by those forward-looking statements are reasonable, the Company can give no assurance that the plans, intentions, expectations or strategies will be attained or achieved. Furthermore, actual results may differ materially from those described in the forward-looking statements and will be affected by a number of risks, uncertainties and assumptions, including, but not limited to, initial and ongoing data from the Company’s preclinical studies and clinical trials not being indicative of final data, the potential size of the target patient populations the Company’s product candidates are designed to treat not being as large as anticipated, the design and success of ongoing and planned clinical trials, future regulatory filings, approvals and/or sales, despite having ongoing and future interactions with the FDA or other regulatory agencies to discuss potential paths to registration for the Company’s product candidates, the FDA or such other regulatory agencies not agreeing with the Company’s regulatory approval strategies, components of the Company’s filings, such as clinical trial designs, conduct and methodologies, or the sufficiency of data submitted, the Company’s pre-commercial activities, commercial launches or operational execution not occurring on anticipated timelines or not supporting planned launches as expected, real-world experience with Attruby/Beyonttra not being consistent with observed biochemical differentiation or not translating into differentiated clinical, commercial or market outcomes, the continuing success of the Company’s collaborations, the Company’s ability to obtain additional funding, including through less dilutive sources of capital than equity financings, potential volatility in the Company’s share price, the Company’s share repurchase program being modified, suspended or discontinued, or share repurchases not delivering the anticipated benefits or proving to be a more attractive use of capital than other alternatives, the impacts of current macroeconomic and geopolitical events, including changing conditions from hostilities in Ukraine and the Middle East, increasing rates of inflation and changing interest rates, on business operations and expectations, as well as those risks set forth in the Risk Factors section of the Company’s most recent Quarterly Report on Form 10-Q and Annual Report on Form 10-K and the Company’s other filings with the U.S. Securities and Exchange Commission. Moreover, the Company operates in a very competitive and rapidly changing environment in which new risks emerge from time to time. These forward-looking statements are based upon the current expectations and beliefs of the Company’s management as of the date of this press release, and are subject to certain risks and uncertainties that could cause actual results to differ materially from those described in the forward-looking statements. Except as required by applicable law, BridgeBio assumes no obligation to update publicly any forward-looking statements, whether as a result of new information, future events or otherwise. BridgeBio Media Contact: Bubba Murarka, Executive Vice President contact@bridgebio.com (650)-789-8220 BridgeBio Investor Contact: Kristen Kelleher, Director of Investor Relations ir@bridgebio.com

2026 年 7 月 14 日至 22 日，FDA 桌上同时摊着五份文件。2 个批准、3 个受理，性质完全不同。 批准是两个：诺华的 Fabhalta 在 IgA 肾病上从加速批准转成了传统批准（2026 年 7 月 16 日），GSK 的 JIDEYTRO 拿到常规批准（7 月 22 日）。 剩下三个只是受理——Apnimed 的口服睡眠呼吸暂停药 AD109（7 月 14 日）、Dyne 的 DMD 外显子 51 跳跃疗法 z-rostudirsen（7 月 20 日）、BridgeBio 的 encaleret（7 月 22 日），PDUFA 目标日分别排在 2027 年 2 月 28 日、2027 年 1 月 21 日、2027 年 5 月 8 日。 三张明年才到期的期票。可这五份文件摊在一张桌上看，比任何一份单独看都有信息量。它们各自代表了一条产业主线，而 2026 年整个行业最要命的那个制度变量，恰好也在这一周露了个头。 一、我把这周的五份文件贴在墙上，然后把表画歪了 先说我为什么会画错。 因为在中文语境里，"FDA 通过了""FDA 点头了""FDA 放行了"这几个说法几乎不分家。而在监管语言里，受理（acceptance for filing）意味着 FDA 认为你交的材料完整到可以开始读，仅此而已。它连"我们觉得这药有戏"都算不上。真正的判决要等 PDUFA 目标日，标准审评是受理后 10 个月，优先审评是 6 个月。 所以这一周严格说是：两个判决 + 三个立案。 我之所以还是把它们摆在一起写，是因为 2026 年的 FDA 已经不是那个"看批准数就够了"的 FDA 了。 先看批准数这条线。 FDA 药品评价与研究中心（CDER）的新分子实体批准数，过去五年是这么走的：2021 年 50 个，2022 年跌到 37 个，2023 年冲到 55 个的高点，2024 年 50 个，2025 年——46 个。 我原本以为 2025 年也是 50。查完之后我把自己稿子里的数字改了：是 46。这个数被 RAPS 和《自然·药物发现评论》分别确认过，其中 34 个新分子实体 + 12 个生物制品，超过一半带孤儿药身份。五年滚动均值因此被拉低到 48 个/年。而 1993 年以来的历史长期均值，是 36 个。 2026 年呢？FDA 官网那张 Novel Drug Approvals 页面最后更新到 7 月 7 日，列了 24 个。加上 7 月 14 日的 Revtorpyk（gedatolisib）和 7 月 16 日的 Lipfendra（enlicitide），到 7 月 24 日大约 26 个。线性外推全年 45 到 48 个。 五年批准数：2023 年的 55 个是高点，2025 年落到 46 个。五年均值被拉低到 48，而 1993 年以来的长期均值只有 36 一年批 46 个还是 50 个，其实差别不大。真正变了的，是这 46 个是怎么被批出来的。  〔术语〕 新分子实体（NME）FDA 统计口径里"真正的新药"——活性成分此前从未在美国获批过。同一个分子换剂型、换适应症、换复方都不算。所以这个数字看着小，但它是衡量一个国家药物创新产出最干净的一把尺子，也是每年年初全行业拿来吵架的那个数。 这一周还有一件事，比五份文件里任何一份都更能说明 2026 年的 FDA 长什么样。7 月 16 日，默沙东的 Lipfendra 获批，全球第一个每日一次口服 PCSK9 抑制剂。它的通用名 enlicitide，在 2025 年 12 月 19 日出现在 FDA 第四批国家优先审评券（CNPV）的名单里。 一张承诺"受理后 1 到 2 个月完成审评"的券。 而同一周，BridgeBio 的 encaleret 拿的是标准审评，PDUFA 排到 10 个月后的 2027 年 5 月 8 日。Apnimed 的 AD109 也是标准审评，2027 年 2 月 28 日。 同一张桌子，两条队。 二、五份文件的准确性质：谁被批了，谁只是排上了队 这一段我要把措辞钉死，因为后面所有推论都建在这张表上。 2026 年 7 月 14–22 日：FDA 桌上的五份文件 日期 药物 / 持有人 监管动作的准确性质 审评类型 关键日期 2026-07-14 AD109（Apnimed） NDA 获受理，未获批 标准审评 PDUFA 2027-02-28 2026-07-16 Fabhalta / iptacopan（诺华） 由加速批准转为传统批准 补充申请 已生效 2026-07-20 z-rostudirsen（Dyne） BLA 获受理，未获批 优先审评 PDUFA 2027-01-21 2026-07-22 JIDEYTRO / zidesamtinib（GSK） 常规批准，非加速批准 RTOR 实时肿瘤学审评 原 PDUFA 2026-09-18，提前约 8 周 2026-07-22 encaleret（BridgeBio） NDA 获受理，未获批 标准审评，不开专家咨询委员会 PDUFA 2027-05-08 这一周的监管时间轴。左侧两个是判决，右侧三个只是立案——中文报道里最常被混为一谈的正是这一点 逐个说清楚。 Fabhalta 那一栏，是这一周最容易被低估的一份文件。  诺华的 iptacopan 在 2024 年 8 月 8 日拿的是 IgA 肾病的加速批准，依据是 9 个月的尿蛋白肌酐比（UPCR）降幅：−44% 对安慰剂 −9%，p<0.0001。 那个适应症的原文措辞是"reduction of proteinuria"——降低蛋白尿，描述的是一个化验值。2026 年 7 月 16 日转正之后，措辞改成了"to slow kidney function decline"——延缓肾功能下降，描述的是临床获益本身。依据换成了 24 个月的 eGFR 总斜率。 这里有个口径问题我得说清楚。 诺华在两份稿子里给了两组略有出入的数字：发表在《新英格兰医学杂志》的版本是 −3.10 对 −6.12 mL/min/1.73m²/年，下降减缓 49.3%；批准当天新闻稿写的是 −3.0 对 −5.7，减缓 48%。 这不是两个独立结论，是同一试验的不同分析集或修约。我采信论文版本，行文里就说"约 48% 到 49%"。至于 95% 置信区间和 p 值，诺华三份新闻稿都没披露。 加速批准给的是一张预付券，传统批准才是把钱结清。Fabhalta 这次结的是两年前那笔账。  〔术语〕 加速批准与传统批准加速批准允许药企拿"合理可能预测临床获益的替代终点"先上市，代价是必须做确证试验。传统（常规）批准要的是临床获益本身或已验证的替代终点。ICER 2026 年 4 月的白皮书统计：1992 到 2021 年间累计授出 278 项加速批准，只有 50% 转正，中位耗时 3.2 年。 JIDEYTRO 那一栏，注意"常规批准"和"二线"这两个词。  zidesamtinib 是 ROS1 阳性非小细胞肺癌的第五款 TKI，2026 年 7 月 22 日获批，走的是常规批准而不是加速批准，还带着突破性疗法认定和孤儿药资格，通过 RTOR 通道比原定 PDUFA 提前了将近两个月。 但它的标签写得很克制，原文是"who received a prior ROS1 kinase inhibitor"——必须是既往用过 ROS1 抑制剂的患者。不含一线。 顺带一句，GSK 是在 2026 年 6 月 9 日宣布以 106 亿美元收购 Nuvalent 的，7 月 15 日交割，7 月 22 日拿到批文。交割后第七天。 z-rostudirsen 那一栏，是这一周唯一的优先审评。  Dyne 在 2026 年 5 月 26 日递交 BLA，7 月 20 日获受理并拿到优先审评，审评期从 10 个月压到 6 个月，PDUFA 2027 年 1 月 21 日。走的是加速批准路径，替代终点是肌肉组织里的 dystrophin 蛋白表达量。确证试验 FORZETTO 三期已经启动，约 90 例，72 周，主终点是"从地面起身的速度"。 encaleret 那一栏有个容易被忽略的利好信号。  BridgeBio 2026 年 5 月 12 日递交，7 月 22 日受理，标准审评，PDUFA 2027 年 5 月 8 日。 公司明确说 FDA 不计划召开专家咨询委员会会议。在 2026 年这个咨询委员会高度政治化的年份里，"不开会"通常意味着审评方对数据没有重大分歧。 〔术语〕 PDUFA 日期与审评绩效PDUFA 是《处方药用户费用法》，药企交审评费，FDA 承诺时限。标准审评 10 个月、优先审评 6 个月（新分子实体各再加 60 天立卷期）。PDUFA 日期不是承诺批准，只是承诺"那天给你个答复"。FDA 每年要向国会报告绩效目标达成情况——这份报告后面会成为本文最硬的一块证据。 制度卡：本周五份文件的性质速览。2 个批准、3 个受理，三张 2027 年才到期的期票（示意图，非官方包装照） 三、暗线一与二：耐药军备竞赛，和口服药正在把注射剂赶出慢病 暗线一：精准肿瘤学已经进入"打耐药"阶段 ROS1 阳性非小细胞肺癌在美国每年大概 3,000 例。这条赛道上已经躺着四款药：克唑替尼（2016 年）、恩曲替尼（2019 年 8 月 15 日）、瑞普替尼 Augtyro（2023 年 11 月 15 日）、他雷替尼 Ibtrozi（2025 年 6 月 11 日）。JIDEYTRO 是第五个。 第五个凭什么进来？ 不是靠缓解率。ARROS-1 注册队列 n=117，全是 TKI 经治患者，数据截止 2025 年 3 月 21 日，客观缓解率 44%（95% CI 34–53），完全缓解只有 0.9%。这个 44% 摆在他雷替尼经治人群 52% 到 62% 的旁边，看着并不占优。 它靠的是另外两组数。 第一组是耐药突变覆盖。 ROS1 抑制剂用久了会长出继发突变，其中最经典的一个叫 G2032R，位置在溶剂前沿。zidesamtinib 在 26 例携带 G2032R 的患者里做到 ORR 54%（95% CI 33–73），缓解持续 6 个月以上的比例 79%、12 个月以上 60%。这是目前该突变上样本量最大的数据。往前一个是瑞普替尼，n=56，38%。更狠的是，用过瑞普替尼的 46 例患者换 zidesamtinib 仍有 41% 缓解，用过他雷替尼的 19 例有 47%。 第二组是耐受性。 这一组数字我看了两遍。zidesamtinib 的头晕发生率 12%，共济失调 2%（全为 1 到 2 级），因不良反应停药率只有 2.3%。同赛道的瑞普替尼，头晕报到 58%。 同一条赛道，头晕发生率差了将近五倍。这不是疗效差异，是脱靶差异——TRK 抑制带来的神经系统副作用 上一代药能杀死肿瘤，但也让一半人天旋地转。这一代药拼的不是杀得更多，是让患者能一直吃下去。  差异从化学上讲得通：克唑替尼、恩曲替尼、瑞普替尼都会一并抑制 TRKA/B/C，头晕、共济失调、味觉异常、体重增加都是这条 off-target 通路的账。zidesamtinib 在 335 个激酶的筛选里，只有 ALK 一个激酶的抑制活性落在 ROS1 IC50 的 10 倍以内。 再看那个"只批二线"的标签。它其实在替前一代药收拾残局——JIDEYTRO 的目标患者，是被克唑替尼、恩曲替尼、瑞普替尼、他雷替尼打过一轮之后耐药、脑转移、无路可走的那批人。 ARROS-1 队列里 49% 有活动性中枢神经系统病灶，中位既往治疗线数 2，最多的一位用过 11 线。颅内客观缓解率 48%，颅内完全缓解 22%。 顺着这条线往前看 2026 年，会发现同样的逻辑在乳腺癌重复了两次： 5 月 1 日获批的 Veppanu（vepdegestrant，一个 PROTAC 降解剂）打的是 ESR1 突变，那正是内分泌治疗耐药的经典机制； 7 月 14 日获批的 Revtorpyk（gedatolisib）打的是 HR+/HER2−、PIK3CA 野生型人群——三联方案中位无进展生存 9.3 个月对氟维司群单药 2.0 个月，客观缓解率 32% 对 1%。 这批人此前没有任何靶向药可用，因为他们"没有可打的突变"。 连"野生型"本身都变成了一个可开发的生物标志物。 暗线二：口服小分子正在把注射剂赶出慢病 这条线在这一周出现了两次，一次是 Fabhalta，一次是 Lipfendra。 Fabhalta 是口服因子 B 抑制剂，作用在补体旁路途径的近端。它的意义要放到 IgA 肾病这条赛道的对手里才看得清：sibeprenlimab 是单抗，atacicept 是皮下注射，ravulizumab 是静脉输注。Fabhalta 是一天两次、200 mg、口服。 〔术语〕 替代终点用一个测得快、测得到的指标去替代"患者活得更久、更好"这种终点。IgA 肾病领域的替代终点从 UPCR（尿蛋白）升级到 eGFR 斜率（肾功能下降速率），Fabhalta 转正走的就是这一级台阶。Dyne 的 DMD 用的是 dystrophin 表达量，那是一级更早、更软的替代终点。 IgA 肾病这条赛道本身也值得记一笔：从 2021 年 12 月 Tarpeyo 加速批准算起，到 2026 年 7 月 7 日 Trutakna 加速批准，四年半，六款药。从"无药可用"到"六个里挑一个"。 但价格是另一回事：Filspari 上市定价 11.88 万美元/年，Tarpeyo 17 万美元/年，Fabhalta 的美国标价 55.0377 万美元/年（这个数来自 ICER 2024 年 3 月 13 日的 PNH 评估报告，是按罕见病适应症锚定的，诺华没有为 IgA 肾病单独定价）。 另一次是 Lipfendra。 2026 年 7 月 16 日获批，全球首个口服 PCSK9 抑制剂，一种大环肽。 CORALreef Lipids 试验 24 周 LDL-C 相对安慰剂降 56%，家族性高胆固醇血症队列降 59%——和注射型 PCSK9 单抗大致相当。月定价 315 美元（折扣前），差不多是现有 PCSK9 产品的一半，分析师给的峰值销售预期 50 亿美元。心血管结局试验还在跑。服药后需要空腹 30 分钟。 一个月 315 美元的口服药，正在给一个原本一年一万多美元、还得自己打针的市场重新定价。  把 2026 年这一整年串起来看，这条线密得有点吓人： 3 月 17 日 Icotyde（icotrokinra），首个口服 IL-23 多肽，打银屑病； 4 月 1 日 Foundayo（orforglipron），口服 GLP-1； 7 月 9 日 Sarclisa 皮下剂型，全身性输注反应从静脉版的 25% 降到 1.5%； 7 月 16 日 Lipfendra。 2026 年"口服/皮下替代注射"的四步。四个月里连出四个，这不是巧合 注射剂在慢病领域正在被系统性地替换掉。不是因为疗效更好，是因为依从性和给药场景。 四、暗线三与四：大众病被药物化，与超罕见病"找不到人" 暗线三：打呼噜从耳鼻喉科和器械店，变成一颗睡前吃的药 Benjafield 等人 2019 年发在《柳叶刀·呼吸医学》上那份全球估算，是这个领域被引最多的锚：30 到 69 岁成人里，轻度到重度阻塞性睡眠呼吸暂停（OSA）患者约 9.36 亿，中度到重度约 4.25 亿。Apnimed 自己的口径更宽，说美国 8,000 万、全球 10 亿——我按论文那个数写。 这么大一个病，此前只有器械没有药。CPAP 是 1980 年代的技术，管用，但轻度 OSA 患者 12 个月的依从率只有 25.7%。 AD109 是 aroxybutynin 加 atomoxetine 的口服复方，每晚睡前一次。SynAIRgy 三期 n=646，26 周。 这里也有口径分歧：公司稿说 AHI 降 55.6%，AJRCCM 论文说降 44.1% 对安慰剂 17.6%，安慰剂校正后的组间差是 −4.0 次/小时（95% CI −6.4 到 −1.6，p=0.001）。我采信论文。剂量也有两个版本，论文写 aroxybutynin 2.5 mg，2026 年 7 月 14 日的受理稿写 2.3 mg，公司没解释为什么改。 这个药最值得琢磨的地方，是 FDA 只给了它标准审评。 一个覆盖 9.36 亿人的病，FDA 给的是标准审评。这个态度本身就是一句评价。  〔术语〕 优先审评券与 CNPV优先审评把 10 个月压到 6 个月。而 2025 年 10 月起启动的国家优先审评券（CNPV，Commissioner's National Priority Voucher）更激进：承诺受理后 1 到 2 个月完成审评，提交前 60 天开始预沟通，不可转让，一般要在两年内用掉。截至 2026 年 5 月 5 日，FDA 分五批共发出 21 张。 暗线四：超罕见病的瓶颈不是研发，是"找到人"和"付得起" encaleret 对的病叫常染色体显性遗传性低钙血症 1 型（ADH1）。 CALIBRATE 三期的数据其实相当漂亮： 67 例随机（encaleret 45 对标准治疗 21），第 24 周同时达到血钙和 24 小时尿钙双达标的比例 76%对 19%，p<0.0001； 甲状旁腺素恢复正常 91.1% 对 0%； 第 3 天就有 71%（32/45）血钙回到正常范围； encaleret 组零停药，97%（65/67）进入长期延展研究。 但真正的瓶颈在别处。ADH1 在美国加欧盟的基因携带者估算约 2.5 万人（约 3.7/10 万），其中只有约两成有明确诊断。 诊断路径长得惊人：低钙血症确诊的中位年龄是 4 岁，拿到 ADH1 基因诊断的中位年龄是 25 岁。中间隔了 21 年。 这 21 年里，患者往往被当成普通甲状旁腺功能减退治疗，而常规补钙加活性维生素 D 恰好会加重尿钙排泄——75% 的患者至少一项并发症，肾钙沉着或肾结石 70%，肾功能损害 57%，基底节钙化 38%。 美国甲状旁腺功能减退症协会的执行主任 Patty Keating 在受理当天的稿子里用了一个词：invisible condition，隐形的疾病。 BridgeBio 给了一个我认为很硬的数字：靠理赔数据统计，从 2023 年 10 月到 2026 年 4 月，美国新确诊 ADH1 患者超过 2,100 人。也就是说，药还没批，找人的工作已经做了两年半。 z-rostudirsen 的分母问题更极端。杜氏肌营养不良（DMD）的发病率是每 3,500 到 5,000 名男婴 1 例，美国约 1.2 万患者，欧盟约 1.6 万。但外显子 51 可跳跃的只占约 13%——美国大约 1,600 例。这 1,600 例里还要扣掉年龄、行走能力、肝肾功能不合适的。 疗效呢？DELIVER 注册扩展队列 32 例（24 例给药 / 8 例安慰剂），6 个月肌肉含量校正后的 dystrophin 表达是 5.46% of normal（P<0.0001），未校正的绝对值 2.87%，相对基线约 7 倍。历史对照 eteplirsen（Exondys 51）大约 0.3%。 5.46%。第一次看到这个数，我的反应是：就这？ 5.46% 的正常人 dystrophin 水平，就是 2026 年 DMD 领域最好的成绩。这不是药不行，是这个病太难。  四条暗线的四个分母：从 9.36 亿人的大众病，到美国 1,600 例的可跳跃亚组 2026 年的宏观底数：这五份文件坐在什么样的地基上 指标 数字 时点 / 口径 CDER 新分子实体批准数 50 / 37 / 55 / 50 / 46 2021 至 2025 逐年 2026 年至今 约26 个；五年均值降至 48，历史长期均值 36 截至 2026-07-24 CNPV 国家优先审评券 已发21 张（5 批），承诺受理后 1–2 个月审评 截至 2026-05-05 FY2025 PDUFA 审评绩效目标 8/10（FY2024 为 10/10） FDA 向国会报告 加速批准转正率 / 中位耗时 50% / 3.2 年；肿瘤适应症撤市 2021 年以来 25 项 1992–2021 累计 278 项 Section 232 医药关税 专利药基准100%；孤儿药、细胞与基因疗法、ADC 为 0% 2026-04-02 公告 IRA 第二轮谈判 15 个药，净省 120 亿美元（−44%） 2027-01-01 生效 孤儿药中位年费用 21.9 万美元对非孤儿药 1.28 万美元 2021 年美元口径 再补两笔制度背景，因为它们直接影响明年那三个 PDUFA。 人事。  FDA 局长 Marty Makary 在 2026 年 5 月 12 日辞职，原食品安全负责人 Kyle Diamantas 出任代理局长。生物制品评价与研究中心（CBER）主任 Vinay Prasad 二进二出——2025 年 7 月 29 日辞职、8 月 9 日回归（隔了 11 天）、2026 年 4 月底第二次离任，起因是 CBER 否决了至少 5 个细胞与基因疗法。 截至 2026 年 7 月 24 日，FDA 由代理局长领导，CBER 主任由副主任代理。裁员规模约 3,500 人。 药价。  2026 年 4 月 2 日的 Section 232 公告把专利药基准关税定在 100%，7 月 31 日对 17 家大型药企生效、9 月 29 日对其余进口商生效。已有 16 家药企签了最惠国（MFN）定价协议，其中 Annex II 的 13 家享 0% 关税至 2029 年 1 月 20 日——但各协议的具体折扣百分比一家都没公开。 TrumpRx 在 2026 年 2 月 5 日上线，实质是个比价导流平台，患者不能在站内买药。IRA 第二轮 15 个药的最高公平价 2025 年 11 月 25 日公布，2027 年 1 月 1 日生效，净省 120 亿美元；第三轮名单 2026 年 2 月 5 日公布，首次纳入 Medicare Part B。 中国部分：1,063 亿美元里，只有 5.8% 是真金 2026 年上半年，中国创新药的 BD 交易总额 1,063 亿美元，约 240 笔。全球最大的十笔 out-licensing 交易里，中国占了 8 席。最大三笔分别是石药集团对阿斯利康 185 亿美元、恒瑞医药对百时美施贵宝 152 亿美元、信达生物对辉瑞 105 亿美元。 同一份统计里还有另一个数：首付款总额 61.58 亿美元。占交易总额的 5.8%。 1,063 亿美元的交易总额里，首付款只有 61.58 亿——占比 5.8% 〔术语〕 license-out 与首付款占比license-out 是把在研药物的海外或全球权益授权给别人开发销售。公布的"总额"通常等于首付款加上研发、注册、销售各阶段的里程碑款，而里程碑要一个个达成才付。首付款占比越低，说明"总额"里的水分越大。2022 年中国交易的平均首付款是 5,200 万美元，到 2026 年升至 1.72 亿美元——绝对值在涨，但占比仍然只有 5.8%。 1,063 亿美元听着像捷报，但真正进账的是 61.58 亿。剩下那 94.2% 要靠未来十年一个个里程碑去兑现。  底子确实厚。ITIF 2026 年 6 月 29 日那份报告给的数：中国占全球在研管线 31.1%（7,141 个药物），美国超过 50%；中国占全球创新药"首次全球批准"的 38%（2015 年是 3%）；ADC 全球管线中国占比超过 42%；2024 年中国肿瘤研究论文 2,600 多篇，首次超过美国。NMPA 2025 年批了 120 个新药，其中 76 个创新药（2024 年是 48 个）。 反面案例也在这一周附近。 2026 年 7 月 10 日，恒瑞医药的 rivoceranib（阿帕替尼）联合 camrelizumab（卡瑞利珠单抗）用于一线不可切除肝细胞癌，收到 FDA 的第三封完整回复函（CRL）。这个 NDA 在 2026 年 1 月 30 日重新受理，PDUFA 原定 2026 年 7 月 23 日——CRL 比 PDUFA 早了 13 天到。三封 CRL，这是中国资产闯 FDA 最典型的挫折样本。 关税那边倒有个意外的中性条款：Section 232 的 0% 豁免清单里明确写了孤儿药、细胞与基因疗法、抗体偶联药物。而中国出海资产高度集中在 ADC 和肿瘤生物药。这是我在整份关税文件里看到的对中国最友好的一行字，而它几乎没人讨论。 五、五个冷问题：这一周暴露的四个矛盾，加一个我自己的判断 写到这儿我得把吹鼓手的帽子摘了。 这一周暴露的四个矛盾。第三条是我的判断，不是事实 第一个，CNPV 正在制造两条队，而这条制度还没经过任何公开检验。 21 张券，五批，最后一批发在 2026 年 5 月 5 日。承诺是受理后 1 到 2 个月完成审评——对比标准审评的 10 到 12 个月，这不是"加快"，这是换了一套流程。 FDA 在 2026 年 3 月 23 日的联邦公报上发了公开听证会通知，6 月 4 日和 6 月 12 日开了两场听证会。也就是说，这个制度已经发了 21 张券、批出了至少六个品种，公众评议才刚刚开始。 问题在于选谁。 券由局长办公室决定，标准写的是"国家优先"，但具体权重怎么算没有公开算法。已知的一条挂钩是：签了 MFN 定价协议的企业，对价里包含关税豁免和 CNPV 券。这就把"你降不降价"和"FDA 多快审你的药"绑在了一起。 我不认为这必然是坏事。 降价换加速，逻辑上说得通。但它意味着审评速度第一次成了一个可以谈判的变量，而不是一个按规则分配的公共资源。 至于 Lipfendra 是不是 CNPV 的产物——PRV Watch 的追踪表把它列进了 2026 年 7 月的 CNPV 获批品种，enlicitide 也确实在 2025 年 12 月 19 日那批券的名单里。 但默沙东官方新闻稿里一个字都没提 CNPV。这两件事之间的因果，截至发稿我没查到官方确认，我只能说"enlicitide 曾获发 CNPV 券"这件事是硬的。 当审评速度可以拿来交换，它就不再是一条规则，而是一件资产。  第二个矛盾更直接：绩效在退步，券还在发。 FDA 自己向国会提交的 PDUFA 财年绩效报告写着：FY2024 达成 10 项审评绩效目标中的 10 项，FY2025 只达成 8 项。这是"审评在退步"最硬的官方证据，因为它出自 FDA 自己的笔。 有意思的是，同一时期 CDER 新药层面的 PDUFA 目标日达标率反而从 2024 年的 94% 升到 2025 年的 96%。两个数字方向相反。 我的读法是：核心新药审评的资源被优先保住了，被挤掉的是那 10 项目标里更边缘的那些——补充申请、会议时限、回函时限之类。3,500 人的裁员，账不会不记。前 CBER 主任对 BMO 分析师说过一句预警：审评中断可能"从 2026 或 2027 年前后开始显现"。 在这样一个绩效已经掉了两项的机构里，继续发出承诺"1 到 2 个月完成审评"的券——多出来的工作量从哪来？ 第三个，这一周五份文件里有三个是受理。我的判断是：2027 年那三个 PDUFA 至少有一个不会顺利。 这句话是我的判断，不是事实，先说清楚。 理由是三条各不相同的软肋。 z-rostudirsen 的替代终点只有 5.46% 的 dystrophin 表达，而当年 CDER 药物评价一办主任 Ellis Unger 就认为 dystrophin 需要达到约 10% 才可能有临床获益； 同一天窗口里，Sarepta 的 Amondys 45 和 Vyondys 53 也在申请把加速批准转成常规批准，PDUFA 2027 年 2 月 28 日——2027 年头两个月是 DMD 监管史上最密集的窗口，FDA 得同时处理三份关于同一个替代终点的判决。 AD109 只拿到标准审评，而它的因不良反应停药率是 21.2% 对安慰剂 3.1%，六个月完成治疗的比例 65.7% 对 83.7%，疲劳量表（PROMIS-Fatigue）没达统计学显著——对一个覆盖上亿人的慢病用药，这组安全性数字会被反复问。encaleret 的样本量只有 67 例随机，虽然不开咨询委员会是个好信号。 第四个，1,063 亿和 61.58 亿之间的那道缝。 前面写过了，5.8%。我再补一层：这个比例是最好的冷静剂，因为它把"中国创新药出海爆发"这个叙事里最不可靠的部分标了出来。里程碑款依赖的是对方公司愿不愿意继续推、临床读出好不好、监管点不点头。恒瑞那第三封 CRL 就是提醒——不是所有里程碑都会到账。 第五个，我这一周最想核实但没核实到的一件事。 大冢制药的 centanafadine（ADHD 适应症，优先审评）PDUFA 目标日是 2026 年 7 月 24 日。这个日期正好落在这一周窗口里。截至发稿我没查到结果。 新闻聚合站对 7 月 15 到 24 日这个时间窗的索引明显滞后两到六周，我搜不到就不瞎猜。同理，我也没能核实这一周是否还有其他我漏掉的批准或受理——所以本文写的是"我确认了的五份文件"，不是"这一周 FDA 全部动作"。 写宏观综述最危险的不是数字错，是把自己没查到的东西写成"没有发生"。  六、把这几个日期记进日历 先给患者和家属一句实话。 这一周对多数人的生活没有任何直接影响。JIDEYTRO 只对既往用过 ROS1 抑制剂的患者开放，Fabhalta 的转正改变的是标签措辞和适用人群门槛（从"快速进展风险"放宽到"进展风险"），另外三个药最早也要到 2027 年才可能上市。 对同行，我认为这一周最该拆的是 GSK 那笔账：106 亿美元买 Nuvalent，交割第七天拿批文，但整个 ROS1 赛道到现在没跑出过一个真正的大品种——Ibtrozi 上市首年净销售只有 2,470 万美元。差异化做得再漂亮，市场天花板不会因此升高。 对中国药企，我认为该盯的不是那 1,063 亿，是首付款结构和 CRL 名单。以及 Section 232 那份 0% 豁免清单——孤儿药、细胞基因疗法、ADC 三项，正好覆盖了中国出海资产最集中的方向。 接下来六个月，这几个日期值得记下来： 2026-07-31，Section 232 关税对 Annex III 列明的 17 家大型药企生效。 2026-09-29，对其余所有进口商生效。 2027-01-01，IRA 第二轮 15 个药的最高公平价正式生效。 2027-01-21，Dyne z-rostudirsen 的 PDUFA。要看的是 FDA 会不会接受 5.46% 这个 dystrophin 水平作为加速批准的依据。 2027-02-28，两件事撞在同一天：Apnimed AD109 的 PDUFA，以及 Sarepta 的 Amondys 45 与 Vyondys 53 从加速批准转常规批准的 PDUFA。一个是新范式的第一道门，一个是老账的清算。 2027-05-08，BridgeBio encaleret 的 PDUFA。全球第一个 ADH1 适应症药物，如果批。 这一周最该记住的一句话 最后两个问题，我自己没有答案，想听你的。 第一个：如果一张券能把审评从 10 个月压到 2 个月，而这张券是靠承诺降价换来的——你觉得这是监管效率的提升，还是监管独立性的折价？ 第二个：AD109 如果在 2027 年 2 月 28 日获批，你会给自己家里那个"打呼噜打得隔两道门都听得见"的人推荐它吗？在知道它 21.2% 的患者会因为不良反应停药之后。 The End 数据来源 1. U.S. FDA, 《Novel Drug Approvals for 2026》, 页面更新 2026-07-07（抓取 2026-07-24）. https://www.fda.gov/drugs/novel-drug-approvals-fda/novel-drug-approvals-2026 2. Nature Reviews Drug Discovery, 《FDA new drug approvals in Q2 2026》, 2026-07-10. https://www.nature.com/articles/d41573-026-00120-7 3. BioSpace, 《Cancer, rare disease dominate FDA approvals in H1 2026 as AstraZeneca, J&J clean up》, 2026-07-09. https://www.biospace.com/fda/cancer-rare-disease-dominate-fda-approvals-in-h1-2026-as-astrazeneca-j-j-clean-up4. Federal Register, 《Commissioner's National Priority Voucher (CNPV) Pilot Program; Public Hearing; Request for Comments》, 2026-03-23. https://www.federalregister.gov/documents/2026/03/23/2026-05573/commissioners-national-priority-voucher-cnpv-pilot-program-public-hearing-request-for-comments5. PRV Watch, 《CNPV Dashboard – Commissioner National Priority Voucher Tracker》, 抓取 2026-07-24（21 张券，5 批，末批 2026-05-05）. https://prvwatch.com/cnpv-dashboard6. Novartis, 《Novartis Fabhalta (iptacopan) receives FDA traditional approval as first and only complement inhibitor to significantly slow kidney function decline in primary IgAN》, 2026-07-16. https://www.novartis.com/news/media-releases/novartis-fabhalta-iptacopan-receives-fda-traditional-approval-first-and-only-complement-inhibitor-significantly-slow-kidney-function-decline-primary-igan7. Novartis, 《Novartis receives FDA accelerated approval for Fabhalta (iptacopan) for reduction of proteinuria in primary IgA nephropathy》, 2024-08-08. https://www.novartis.com/news/media-releases/novartis-receives-fda-accelerated-approval-fabhalta-iptacopan-first-and-only-complement-inhibitor-reduction-proteinuria-primary-iga-nephropathy-igan8. ICER, 《Final Evidence Report on Treatments for Paroxysmal Nocturnal Hemoglobinuria》（Fabhalta 美国标价 550,377 美元/年）, 2024-03-13. https://icer.org/news-insights/press-releases/institute-for-clinical-and-economic-review-publishes-final-evidence-report-on-treatment-for-paroxysmal-nocturnal-hemoglobinuria/9. Apnimed, 《Apnimed Announces FDA Acceptance of New Drug Application for AD109》, 2026-07-14（PDUFA 2027-02-28，标准审评）. https://apnimed.com/article/apnimed-announces-fda-acceptance-of-new-drug-application-for-ad109-an-investigational-oral-pill-to-treat-adults-with-obstructive-sleep-apnea/10. American Journal of Respiratory and Critical Care Medicine, 《Phase 3 SynAIRgy Trial of AD109 in Obstructive Sleep Apnea》（PMID 42148495）, 2026-05-18. https://www.prnewswire.com/news-releases/apnimed-announces-publication-of-its-phase-3-synairgy-trial-of-ad109-for-obstructive-sleep-apnea-in-the-american-journal-of-respiratory-and-critical-care-medicine-302774259.html11. Benjafield AV et al., 《Estimation of the global prevalence and burden of obstructive sleep apnoea》, The Lancet Respiratory Medicine 2019;7(8):687-698, 2019-07. https://pubmed.ncbi.nlm.nih.gov/31300334/12. Dyne Therapeutics, 《Dyne Therapeutics Announces U.S. FDA Acceptance of Biologics License Application (BLA) for Z-Rostudirsen in Exon 51 Duchenne Muscular Dystrophy》, 2026-07-20. https://www.globenewswire.com/news-release/2026/07/20/3329635/0/en/dyne-therapeutics-announces-u-s-fda-acceptance-of-biologics-license-application-bla-for-z-rostudirsen-in-exon-51-duchenne-muscular-dystrophy-dmd.html13. Dyne Therapeutics, 《Announces Submission of BLA to U.S. FDA for Z-Rostudirsen》, 2026-05-26. https://www.globenewswire.com/news-release/2026/05/26/3300969/0/en/Dyne-Therapeutics-Announces-Submission-of-Biologics-License-Application-BLA-to-U-S-FDA-for-Z-Rostudirsen-in-Exon-51-Duchenne-Muscular-Dystrophy-DMD.html14. BioPharm International, 《FDA Accepts sNDAs for Sarepta's Amondys 45 and Vyondys 53, Targeting Traditional Approval in Duchenne》（PDUFA 2027-02-28）, 2026-06-30. https://www.biopharminternational.com/view/fda-accepts-sndas-for-sarepta-s-amondys-45-and-vyondys-53-targeting-traditional-approval-in-duchenne15. BridgeBio, 《BridgeBio Announces FDA Acceptance of NDA for Encaleret for ADH1》, 2026-07-22（PDUFA 2027-05-08，标准审评，不开 AdCom）. https://www.globenewswire.com/news-release/2026/07/22/3331254/0/en/BridgeBio-Announces-FDA-Acceptance-of-NDA-for-Encaleret-for-ADH1.html16. BridgeBio, 《BridgeBio Submits NDA to FDA for Encaleret》（CALIBRATE 三期数据）, 2026-05-12. https://www.stocktitan.net/news/BBIO/bridge-bio-submits-nda-to-fda-for-encaleret-for-individuals-living-kqyu09arictt.html17. GSK / Nuvalent, 《GSK enters agreement to acquire Nuvalent, Inc.》（每股 124 美元，总价 106 亿美元）, 2026-06-09. https://investors.nuvalent.com/2026-06-09-GSK-enters-agreement-to-acquire-Nuvalent,-Inc18. Fierce Pharma, 《GSK secures first-ever lung cancer FDA approval with Jideytro from Nuvalent deal》（含 Ibtrozi Q1 2026 销售 1,850 万美元）, 2026-07-22. https://www.fiercepharma.com/pharma/gsk-secures-first-ever-lung-cancer-fda-approval-jideytro-nuvalent-deal19. Nuvalent, 《ARROS-1 registrational cohort data in TKI pre-treated ROS1-positive NSCLC》（数据截止 2025-03-21）, 2025-06-24. https://investors.nuvalent.com/news-releases20. Merck, 《Merck's LIPFENDRA (enlicitide) is the First and Only Once-Daily Oral PCSK9 Inhibitor Approved by the U.S. FDA》, 2026-07-16. https://www.merck.com/news/mercks-lipfendra-enlicitide-is-the-first-and-only-once-daily-oral-pcsk9-inhibitor-approved-by-the-u-s-fda-to-reduce-ldl-c-in-adults-with-hypercholesterolemia/21. Fierce Pharma, 《Merck scores FDA nod; Lipfendra becomes first oral PCSK9 treatment》（月定价 315 美元、峰值预期 50 亿美元）, 2026-07-16. https://www.fiercepharma.com/pharma/merck-scores-fda-nod-lipfendra-becomes-first-oral-pcsk9-treatment22. Drugs.com, 《FDA Approves Revtorpyk (gedatolisib) for HR+/HER2−, PIK3CA Wild-Type Advanced Breast Cancer》, 2026-07-14. https://www.drugs.com/newdrugs/fda-approves-revtorpyk-gedatolisib-hr-her2-pik3ca-wild-type-locally-advanced-metastatic-breast-6838.html23. ICER, 《Strengthening the FDA's Accelerated Approval Pathway》（白皮书）, 2026-04. https://icer.org/wp-content/uploads/2026/04/Strengthening-the-FDAs-Accelerated-Approval-Pathway-_-ICER-White-Paper-_-April-2026.pdf24. STAT, 《FDA Commissioner Marty Makary resigns, replaced by Kyle Diamantas》, 2026-05-12. https://www.statnews.com/2026/05/12/fda-commissioner-marty-makary-resigns-kyle-diamantas-acting/25. STAT, 《FDA's Vinay Prasad, controversial CBER chief, to depart》（CBER 否决至少 5 个细胞与基因疗法）, 2026-03-06. https://www.statnews.com/2026/03/06/fda-vinay-prasad-controversial-cber-director-leaving/26. Fierce Pharma, 《Former CBER director warns FDA staff cuts could upend drug review timelines as early as next year: BMO》, 2026-01（具体日期原页未标注）. https://www.fiercepharma.com/pharma/recently-departed-cber-director-warns-fda-staff-cuts-could-upend-drug-review-timelines-early27. Ropes & Gray, 《100% On Brand: U.S. Imposes New Tariffs (and Key Exemptions) on Patented Pharmaceuticals》（孤儿药、细胞基因疗法、ADC 为 0%）, 2026-04. https://www.ropesgray.com/en/insights/alerts/2026/04/100-on-brand-us-imposes-new-tariffs-and-key-exemptions-on-patented-pharmaceuticals28. Pharmacy Times, 《Roundup: Every Most Favored Nation Agreement With the Trump Administration》（16 家已签，更新日）, 2026-07-24. https://www.pharmacytimes.com/view/round-up-every-most-favored-nation-agreement-with-the-trump-administration29. American Action Forum, 《Inflation Reduction Act IPAY 2027 Maximum Fair Prices Have Been Published》, 2025-11-25. https://www.americanactionforum.org/insight/inflation-reduction-act-ipay-2027-maximum-fair-prices-have-been-published/30. American Action Forum, 《CMS' Third IRA Negotiation List: Selections, Signals, and Potential Savings》, 2026-02-05. https://www.americanactionforum.org/insight/cms-third-ira-negotiation-list-selections-signals-and-potential-savings/31. Pharmacy Times, 《TrumpRx Launches, Offering Cash-Paying Patients Discounted Drugs》, 2026-02-05. https://www.pharmacytimes.com/view/trumprx-launches-offering-cash-paying-patients-discounted-drugs32. Evaluate, 《Evaluate Releases 2026 Orphan Drug Report》（2032 年孤儿药销售额超 4,000 亿美元）, 2026-03-12. https://www.evaluate.com/press-release/2026-orphan-drug-report/33. Optum, 《Orphan drugs are sweeping the market — but can we afford them?》（孤儿药中位年费用 21.9 万美元，2021 年美元口径）, 2023-05-18. https://business.optum.com/en/insights/orphan-drugs-market-can-we-afford-them.html34. ITIF, 《China's Burgeoning Biopharmaceutical Competitiveness Demands a US Response》（管线 31.1%、首发批准 38%、ADC 超 42%）, 2026-06-29. https://itif.org/publications/2026/06/29/chinas-burgeoning-biopharmaceutical-competitiveness-demands-us-response/35. BigGo Finance（数据源 VBData）, 《China's Innovative Drug BD Deals Top $100 Billion in H1 2026, Claiming 8 of Global Top 10 Spots》（总额 1,063 亿美元、首付款 61.58 亿美元）, 2026-07-15.（中文相关数据的英文可引用源）https://finance.biggo.com/news/5f16a5c6-38b3-4a57-87cd-b1a63daa090236. Elevar Therapeutics, 《Elevar Therapeutics Receives Complete Response Letter from FDA for Rivoceranib + Camrelizumab》（恒瑞资产第三封 CRL）, 2026-07-10.（中文相关事件的英文一手源）https://elevartx.com/2026/07/10/elevar-crl-july-2/37. SCMP, 《China's biotech boom drives surge in home-grown innovative drug approvals》（NMPA 2025 年 76 个创新药）, 2026-05-27.（中文来源的英文报道）https://www.scmp.com/business/china-business/article/3355021/chinas-drug-regulator-clears-wave-home-grown-innovative-medicines-amid-biotech-boom38. HHS, 《WTAS: FDA Launches Framework for Accelerating Development of Individualized Therapies for Ultra-Rare Diseases》, 2026-02-24. https://www.hhs.gov/press-room/wtas-fda-launches-framework-accelerating-therapies-ultra-rare-diseases.html 免责声明 本文仅为医药行业信息分析与科普，不构成任何诊疗建议、用药指导或投资建议。 文中涉及的药物均为处方药，是否适用请以主治医师判断及所在国家/地区监管机构批准的说明书为准。 文中数据均引自公开来源，如与官方文件不一致，以官方文件为准。 文中对 2027 年三个 PDUFA 结果的推测，以及对 CNPV 制度影响的判断，均为作者个人观点，不代表任何机构立场。 原创声明 本文为原创内容，未经授权禁止转载、摘编或洗稿。 转载请后台联系获取授权，授权后须完整保留作者与出处。 如果觉得内容还不错，别忘了点 分享、 收藏、 在看、 点赞 哦！