A Multicenter, Open-Label, Uncontrolled, Single Arm Clinical Trial to Evaluate the Efficacy and Safety of ACP-015 Administered Subcutaneously Once Weekly for 52 Weeks in Children with Achondroplasia Aged 2 to 11 Years.

开始日期2025-10-01

申办/合作机构

Teijin Pharma Ltd.

NCT06732895

/ Recruiting临床2期

A Phase 2b, Multicenter, Double-Blind, Randomized, Placebo-controlled Trial Evaluating Efficacy and Safety of Subcutaneous Doses of Navepegritide Administered Once Weekly for 52 Weeks in Adolescents (12-18 Years of Age) With Achondroplasia.

The purpose of this clinical trial is to evaluate efficacy and safety of once weekly subcutaneous (SC) doses of navepegritide 100 μg/kg compared to placebo (inactive drug) in adolescents aged 12 to 18 years with Achondroplasia. What will be measured is Annualized Growth Velocity after a 52-week treatment period.

开始日期2024-12-10

申办/合作机构

Ascendis Pharma A/S

NCT06433557

/ Active, not recruiting临床2期

A Phase 2, Open-Label, Single-Arm, 156-week Trial to Investigate the Efficacy, Safety and Tolerability of Combined Once Weekly Navepegritide and Lonapegsomatropin in Children With Achondroplasia

This proof-of-concept trial is being conducted to evaluate the efficacy, safety and tolerability of combination treatment with navepegritide and lonapegsomatropin administered as separate subcutaneous (SC) injections once weekly in children with achondroplasia (ACH) aged 2 to 11 years.

开始日期2024-07-26

申办/合作机构

Ascendis Pharma Growth Disorders A/S

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100 项与那韦培肽相关的专利（医药）

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项与那韦培肽相关的文献（医药）

2026-06-03·Journal of the Endocrine Society

Achondroplasia management in the era of targeted therapies: a meta-analysis of C-type natriuretic peptide analogs

Review

作者: Uday, Suma ; Ashraf, Ambika P ; Pappachan, Joseph M ; Dutta, Deep ; Nagendra, Lakshmi ; Kamrul-Hasan, Abul Bashar Mohammad ; Bhattacharya, Saptarshi

Abstract:

Context:

No prior meta-analysis has systematically assessed efficacy and safety of C-type natriuretic peptide (CNP) analogs within the context of evolving understanding of FGFR3 biology and achondroplasia natural history.

Objective:

To evaluate the safety and efficacy of CNP analogs in children with achondroplasia and contextualize clinical outcomes and natural history.

Methods:

Systematic review of randomized control trials and real-world studies evaluating the safety and efficacy of CNP analogs (vosoritide and navepegritide) in children aged <18 years with genetically confirmed achondroplasia was performed. Coprimary outcomes of interest were adverse events (AEs) and changes from baseline in annualized growth velocity (AGV) at the end of the trials. Secondary outcomes included changes from baseline in height Z-score, standing height, and upper-to-lower body segment (ULS) ratio.

Results:

Eleven studies (N = 542) were included, of which 4 RCTs (n = 326) with low overall risk of bias were meta-analyzed. Overall and serious AE rates were comparable between CNP analogs and placebo, except for higher relative risks of injection site reactions (1.65), urticaria (4.04), and swelling (3.57). C-type natriuretic peptide analogs significantly increased mean differences in AGV (1.36 cm/year; 95% CI: 1.05-1.68; P < .00001) and standing height (1.24 cm; 95% CI: 0.47-2.01; P = .002), without short-term effect on ULS ratio. Real-world studies demonstrated sustained growth benefits with infrequent serious AEs or treatment discontinuations.

Conclusion:

C-type natriuretic peptide analogs provide slight but statistically meaningful improvements in linear growth in children with achondroplasia with acceptable short-term safety profile. Long-term studies are needed to define optimal timing of therapy on adult height, functional outcomes, and achondroplasia-related complications.

2026-06-01·EUROPEAN JOURNAL OF ENDOCRINOLOGY

Navepegritide combined with lonapegsomatropin for the treatment of children with achondroplasia: 52-week results from the phase 2 COACH trial

Article

作者: Dalby, Lene Worsaae ; Ominsky, Michael S ; Jensen, Simon Mejlhede ; Nolting, Line Aagaard ; Irving, Melita ; Komirenko, Allison S ; McDonnell, Ciara M ; Iklé, Jennifer M ; Abdelrahman, Sohair G ; Hove, Hanne B ; Shu, Aimee D

Abstract:

Objective:

Navepegritide, a prodrug of C-type natriuretic peptide, has demonstrated improved growth and additional benefits, including lower limb alignment, in children with achondroplasia. The well-described mechanism of action of navepegritide in the growth plate is hypothesized to enhance its receptivity to lonapegsomatropin, a prodrug of somatropin, to provide benefits beyond those seen with monotherapies in achondroplasia.

Methods:

In the proof-of-concept phase 2 COACH trial, children with achondroplasia aged 2 to 11 received weekly navepegritide and lonapegsomatropin. The primary efficacy endpoint was annualized growth velocity (AGV) at week 52 vs AGV with navepegritide monotherapy in the ApproaCH trial (NCT05598320).

Results:

Twenty-one children were enrolled: 12 received no prior growth-promoting treatment (treatment-naive, TN) and 9 had been receiving navepegritide monotherapy for >1 year (navepegritide-experienced, NE). At week 52, TN children receiving navepegritide and lonapegsomatropin had a least squares mean AGV of 8.69 cm/year versus 5.95 cm/year for matched children who received navepegritide monotherapy, a difference of 2.74 cm/year (P < .0001). Observed mean AGV for TN children was 8.80 cm/year, a +3.89 cm/year increase from baseline (P = .0015). NE children had an observed mean AGV of 8.42 cm/year, a +3.28 cm/year increase from navepegritide-treated baseline (P < .0001). An improvement in body proportionality was observed and arm span increased substantially (+9.4 cm in TN and +7.9 cm in NE children), in line with gains in height. Treatments were well-tolerated with generally mild adverse events and no treatment discontinuations.

Conclusions:

In this prospective trial, the robust growth and anthropometric results suggest that by continuously relieving the pre-hypertrophic block in achondroplasia, navepegritide may make the growth plate more responsive to the strong complementary effect of lonapegsomatropin.

Trial registration details:

ClinicalTrials.gov NCT06433557

2026-01-01·JAMA Pediatrics

Once-Weekly Navepegritide in Children With Achondroplasia

Article

作者: de Bergua Domingo, Josep Maria ; Hove, Hanne B. ; Savarirayan, Ravi ; Legare, Janet M. ; Bacino, Carlos A. ; Mao, Meng ; McDonnell, Ciara ; Smit, Kevin ; Hoernschemeyer, Daniel G. ; Smith, Alden ; Shu, Aimee D. ; Hofman, Paul L. ; Freiberg, Lærke C. ; Abuzzahab, M. Jennifer ; Ominsky, Michael S. ; Campeau, Philippe M. ; Ward, Leanne M.

Importance:

Historically considered a skeletal dysplasia characterized by disproportionate short stature, achondroplasia is a condition with multisystemic effects due to the widespread expression of the fibroblast growth factor receptor 3 variant throughout the body, impacting muscle, neurological function, cardiorespiratory health, and health-related quality of life.

Objective:

To evaluate the efficacy, safety, and tolerability of once-weekly navepegritide, an investigational prodrug of C-type natriuretic peptide, while assessing benefits beyond growth that may have important implications for complications and health-related quality of life in children with achondroplasia.

Design, Setting, and Participants:

Enrollment for this pivotal phase 2b, randomized, double-blind, placebo-controlled trial (APPROACH) was conducted between March and August 2023 at 10 hospitals in Australia, Canada, Denmark, Ireland, New Zealand, Spain, and the US with randomized, blind treatment through 52 weeks and an open-label extension (ongoing). Eligible participants aged 2 to 11 years had achondroplasia confirmed by genetic testing, were naive to treatment with growth-promoting agents, and had their height recorded at least 6 months prior to randomization. Enrolled participants were stratified by age and sex. Those with radiographic evidence of closed growth plates, planned bone surgery, severe untreated sleep apnea, or medical conditions known to affect growth were excluded (n = 2 of 86); of 84 participants enrolled, all were analyzed for safety and efficacy outcomes, including 2 who discontinued treatment.

Interventions:

Navepegritide (100 μg/kg/wk) or placebo administered by once-weekly subcutaneous injection.

Main Outcomes and Measures:

The primary end point was annualized growth velocity at week 52. Other clinically important secondary measures included radiographically assessed skeletal outcomes and health-related quality of life, evaluated using Achondroplasia Child Experience Measures. Safety assessments included adverse events, clinical laboratory assessments, bone age, and immunogenicity.

Results:

Eighty-four participants were enrolled and assigned randomly in a 2:1 ratio to receive navepegritide (n = 57; mean [SD] age, 5.6 [2.6] years; 31 [54%] male) or placebo (n = 27; mean [SD] age, 6.0 [2.7] years; 14 [52%] male). All randomized participants were included in efficacy and safety analyses, although 2 patients in the navepegritide group discontinued treatment (one at week 26 and the other at week 34). The trial met its primary end point, demonstrating superiority of navepegritide in annualized growth velocity at week 52 vs placebo (least-squares mean treatment difference of 1.49 cm/y; 95% CI, 1.05 to 1.93; P &lt; .001). Treatment resulted in improvements (least-squares mean treatment difference [95% CI]) in tibial-femoral angle (−1.81° [−3.16 to −0.47]), mechanical axis deviation (−2.78 mm [−4.71 to −0.86]), fibula to tibia length ratio (−0.016 [−0.024 to −0.008]), and Achondroplasia Child Experience Measures–Physical Functioning (−11.1 [−21.5 to −0.80] in children younger than 5 years). No serious adverse events were treatment-related, and no deaths occurred. Injection site reaction rates were low, and no symptomatic hypotension or fractures were observed.

Conclusions:

In this randomized clinical trial, navepegritide treatment resulted in statistically significantly higher annualized growth velocity in children with achondroplasia, with a similar safety and tolerability profile vs placebo. Moreover, navepegritide demonstrated additional potential health benefits beyond growth.

Trial Registration:

ClinicalTrials.gov Identifier: NCT05598320

191

项与那韦培肽相关的新闻（医药）

2026-08-10

·BioSpace

BioMarin axes asset from $270M Inozyme takeover, ending run in rare metabolic indication

iStock, drogatnev BioMarin’s discontinued drug showed biomarker benefits in a Phase 3 study for ENPP1 deficiency but these failed to translate to clinical improvements. BioMarin will no longer invest in an investigational enzyme replacement therapy following a disappointing late-stage showing earlier this year. The asset, dubbed BMN 401, was being studied in the Phase 3 ENERGY trial for ENPP1 deficiency , a rare metabolic disease that involves a range of symptoms, including early-onset osteoporosis, the calcification of tendons and a higher risk of cardiovascular death. The candidate came to BioMarin in May 2025 as the centerpiece of its $270 million takeover of Inozyme Pharma. A year later, however, the asset turned out to be a disappointment, meeting only one of two key endpoints in ENERGY. While BMN 401 significantly lowered levels of a key disease marker, this effect failed to translate into clinical benefit for patients. ezstandalone.cmd.push(function () { ezstandalone.showAds(129); }); The ENERGY failure has prompted BioMarin to discontinue the development of BMN 401 across all indications, according to the company’s second quarter earnings report released on Thursday. In addition to ENPP1 deficiency, Inozyme had previously been testing the molecule for ABCC6 deficiency and calcific uremic arteriopathy, both rare diseases. BioMarin no longer lists any active program in these indications. Rare diseases BioMarin suffers another blow to rare disease portfolio in Phase 3 flop BioMarin’s investigational therapy failed to elicit clinical improvements in patients with ENPP1 deficiency, while also missing key secondary endpoints of rickets severity and growth. May 19, 2026 · 2 min read · Tristan Manalac Read more Other than the discontinuation, BioMarin reported positive earnings results this quarter. Revenues climbed 20% year-on-year to hit $990 million, the biopharma reported. BioMarin’s product portfolio was led by the achondroplasia medicine Voxzogo, which brought in $253 million. Of note, Voxzogo sales continued to grow—14% versus the same period in 2025—despite the entry of a key competitor: Ascendis Pharma’s Yuviwel, which won FDA approval in achondroplasia, the most common cause of dwarfism, this past March. Ascendis is scheduled to present its second quarter earnings on Aug. 13. “Voxzogo continues to offset any impact with growth in the <2 [years] population and in the ex-US population, which comprises the majority of revenues,” Stifel analysts wrote in a note on Friday. “The next couple quarters will be key here in determining the competitive impact to BioMarin.” In October last year, in anticipation of Yuviwel’s approval, BioMarin withdrew a previously announced goal of hitting $4 billion in revenue by 2027—a decision driven largely by the impact of potential challengers to Voxzogo. On Thursday, however, BioMarin’s confidence in Voxzogo appeared to grow, raising full year sales guidance from a range of $975 million to $1.02 billion to a minimum of $1 billion with the potential to reach $1.05 billion. Aside from Ascendis, BridgeBio Pharma is also advancing on the achondroplasia market with its investigational pill dubbed infigratinib . Data released in late June showed that aside from boosting growth in children, infigratinib also improved body proportionality , a finding that could help the drug stand out in the achondroplasia arena. A regulatory filing for infigratinib is planned for the third quarter, with a potential launch in early to mid-2027. Earnings BioMarin Abandons Gene Therapy Roctavian, Cuts Revenue Guidance as Voxzogo Faces Competitors BioMarin no longer expects to hit its $4 billion revenue target by 2027, citing various market factors such as impending competition for achondroplasia therapy Voxzogo and the divestment of the gene therapy Roctavian. October 28, 2025 · 4 min read · Tristan Manalac Read more

临床3期上市批准并购临床结果基因疗法

2026-08-06

·GlobeNewswire-Health Care

Ascendis Announces COACH Week 78 Results and Provides Update on Achondroplasia Programs and YUVIWEL® Uptake in the U.S.

TransCon® CNP and TransCon hGH combination therapy Week 78 data from the Phase 2 COACH Trial showed durable, unprecedented increase in healthy, proportional growthTransCon CNP monotherapy Week 104 data from the pivotal ApproaCH Trial demonstrated durable improvements in height, body proportionality, and lower-limb alignmentCompleted target enrollment for pivotal reACHin Trial supporting planned regulatory filings for infants 0 to <2 years of age with achondroplasiaSafety and tolerability of monotherapy and combination therapy remained consistent with previously reported dataMore than 170 unique YUVIWEL patient enrollments in the U.S., with more than 65% approved for reimbursement, through June 30 COPENHAGEN, Denmark, Aug. 06, 2026 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (Nasdaq: ASND) today provided updates across its achondroplasia programs. “The rapid uptake of YUVIWEL in the United States underscores its highly differentiated profile and reflects our core values and our patient-centered development of therapies,” said Jan Mikkelsen, President and Chief Executive Officer of Ascendis Pharma. “We are very pleased to see continued unprecedented results from our combination therapy trial, which further reinforce our commitment to ensure that as many children as possible can access YUVIWEL.” Combination Therapy Update (TransCon CNP + TransCon hGH; navepegritide + lonapegsomatropin) Treatment with the combination of once-weekly TransCon CNP and once-weekly TransCon hGH continued to demonstrate durable growth in children with achondroplasia, with a mean annualized growth velocity (AGV) meeting or exceeding the 97th percentile of children of average stature, without compromising safety or tolerability at Week 78. Highlights of the Topline Week 78 COACH Trial Results Consistent with Week 26 and Week 52 results, mean AGV for children treated with combination therapy through Week 78 remained at or above the 97th percentile AGV of children of average stature, with changes over time following age-related growth patterns, and sustained increases in ACH height Z-score, indicating a tripling of efficacy compared to TransCon CNP monotherapy. For the TransCon CNP treatment-naïve cohort, mean AGV at Week 78 was 7.73 cm/year, with an increase in mean ACH height Z-score of +1.29, increasing from 0.46 to 1.75 over 78 weeks.For the TransCon CNP-experienced cohort (mean treatment duration with TransCon CNP of 2.56 years), mean AGV at Week 78 was 7.67 cm/year, with an improvement in mean ACH height Z-score of +1.10, increasing from 1.28 to 2.38 over 78 weeks. Children treated with combination therapy demonstrated continued improvements in body proportionality through Week 78, aligning with the increase in linear growth.Safety and tolerability were consistent with those observed for TransCon CNP and TransCon hGH monotherapies. Combination therapy was generally well-tolerated, with a low incidence of injection site reactions and generally mild treatment-emergent adverse events (TEAEs).To date, 100% of the 21 enrolled children completed 78 weeks of treatment and remain on therapy in the COACH Trial.Additional data from Week 78 of COACH to be presented at an upcoming medical meeting. COACH Trial DesignCOACH is an ongoing prospective Phase 2 open-label trial to investigate the efficacy, safety, and tolerability of combined treatment with once-weekly TransCon CNP at 100 µg/kg/week and once-weekly TransCon hGH at a starting dose of 0.30 mg/kg/week (“combination therapy”) in children with achondroplasia aged 2 to 11 years. The trial included a cohort of TransCon CNP treatment-naïve children (N=12, mean age 5.26 years) and a cohort of previously TransCon CNP-treated children (N=9, mean age 8.32 years), who had received TransCon CNP (100 µg/kg/week) for a mean of 2.56 years in clinical trials. The trial population is representative of children with achondroplasia and the prior treatment benefits of TransCon CNP monotherapy. “I became involved in advocacy in part because parents were increasingly eager to learn more about emerging drug development programs,” said Chandler Crews, Founder of The Chandler Project. “YUVIWEL is a new treatment option that has brought hope to many in our community seeking to prevent complications of achondroplasia that may, without effective pharmacologic treatment, lead to chronic pain, mobility issues, surgeries, and impact on quality of life.” Monotherapy Update(TransCon CNP; navepegritide) In completed and ongoing clinical trials of children with achondroplasia, treatment with once-weekly TransCon CNP monotherapy demonstrated durable improvements in height, as well as benefits beyond height, and a safety and tolerability profile similar to placebo, including a low rate of injection site reactions, consistent with the incidence rate reported in the FDA label. Week 104 data from the pivotal ApproaCH Trial of TransCon CNP at 100 µg/kg once-weekly in children with achondroplasia aged 2 to 11 years demonstrated: Durable improvements in height as measured by AGV and height Z-scores over 104 weeks for those randomized to TransCon CNP, and significant improvement for those switching from placebo to TransCon CNP at Week 52.Benefits beyond height with TransCon CNP monotherapy, including improvements in lower limb alignment and body proportionality, as demonstrated by improved tibial-femoral angle and upper-to-lower body segment ratio, respectively, with durable treatment effect over 104 weeks for those randomized to TransCon CNP, and substantial improvement at Week 104 for those switching from placebo to TransCon CNP at Week 52.Through two years of treatment, TransCon CNP was generally well-tolerated. Most adverse events in TransCon CNP-treated children were mild or moderate, with none leading to treatment discontinuation or withdrawal from the trial. There were no occurrences of symptomatic hypotension, and the overall rate of injection-site reactions, all of which were mild, was 0.35 per person-year of exposure. Completed target enrollment for pivotal reACHin Trial, supporting planned regulatory filings for infants 0 to <2 years of age with achondroplasia.To date, 96% of the 140 children enrolled in the AttaCH long-term open-label extension trial remain on TransCon CNP monotherapy, with up to nearly 6 years of treatment, or are in the ongoing COACH combination therapy trial. The AttaCH Trial, following children to near-final adult height, continues to enroll children who complete TransCon CNP monotherapy trials (ACcomplisH, ApproaCH, teACH, and reACHin).The ongoing pivotal reACHin Trial is evaluating the safety, tolerability, and efficacy of TransCon CNP in infants with achondroplasia (aged 0 to <2 years).The ongoing pivotal teACH Trial is evaluating the safety, tolerability, and efficacy of TransCon CNP in adolescents with achondroplasia (aged 12 to <18 years). In the European Union, a decision is anticipated in the fourth quarter of 2026 for the Marketing Authorisation Application for TransCon CNP as a monotherapy for children with achondroplasia. “I am excited to see compelling long-term efficacy, safety, and tolerability data for TransCon CNP monotherapy,” said Carlos Bacino, MD, FACMG, Professor of Molecular and Human Genetics, Baylor College of Medicine and Texas Children’s Hospital. “TransCon CNP has demonstrated positive effects compared to placebo on multiple aspects of skeletal growth, including statistically significant improvements in height and lower-limb alignment, with growing long-term data highlighting its unprecedented efficacy when used in combination with TransCon hGH. That and its once-weekly administration and low rate of injection site reactions mark it as an important potential new treatment option for children with achondroplasia.” “We are encouraged to see research on navepegritide continue to examine areas the achondroplasia community has identified as important, including outcomes beyond linear growth,” said Mike Hughes, Chair, Biotech Industry Liaison Committee, Little People of America. “The ApproaCH findings add to our understanding of body proportionality and lower-limb alignment, while continued study will be important to determine whether these anatomical changes translate into meaningful differences in function, mobility, or the future need for surgical intervention. Clear, balanced evidence can help individuals and families make informed decisions aligned with their own goals and values, including whether treatment is right for them.” YUVIWEL U.S. Launch Update through June 30, 2026(navepegritide; developed as TransCon CNP) More than 170 unique patient enrollments by approximately 90 prescribing healthcare providers, with more than 65% approved for reimbursement, through June 30, 2026.All enrollments through this date reflect patients new to YUVIWEL therapy. A slide presentation with these updates will be made available on the Investors & News section of the Ascendis Pharma website: https://investors.ascendispharma.com. About TransCon CNP and TransCon hGHTransCon CNP (navepegritide) is a prodrug of C-type natriuretic peptide (CNP) administered once weekly, designed to provide continuous exposure of active CNP to receptors on tissues throughout the body to counteract the overactive FGFR3 signaling in achondroplasia. TransCon hGH is a prodrug of somatropin administered once weekly, providing sustained release of active, unmodified somatropin. In February 2026, TransCon CNP was approved by the U.S. Food & Drug Administration (FDA) under the trade name YUVIWEL® to increase linear growth in pediatric patients 2 years of age and older with achondroplasia with open epiphyses. TransCon hGH (lonapegsomatropin) is investigational in achondroplasia and other indications and is approved and marketed as SKYTROFA® for the treatment of pediatric and adult growth hormone deficiency. About AchondroplasiaAchondroplasia is a rare genetic condition arising from a systemic fibroblast growth factor receptor 3 (FGFR3) variant that leads to an imbalance in the effects of the FGFR3 and CNP signaling pathways, estimated to affect more than 250,000 people worldwide. While historically considered a bone growth disorder, the FGFR3 variant seen in achondroplasia is expressed in tissues throughout the body, and is associated with an increased risk of muscular, neurological, and cardiorespiratory complications in addition to skeletal dysplasia. Medical complications of achondroplasia can vary from individual to individual and across different stages of life. Throughout infancy and childhood, observed complications include spinal abnormalities, enlarged brain ventricles, impaired muscle strength and reduced stamina, hearing deficits and chronic ear infections, upper airway obstructions, sleep-disordered breathing, hip problems, leg bowing, and chronic pain; some of which persist or worsen in adulthood. These medical complications can affect physical well-being and quality of life, and may be impacted by a range of individual, clinical, and social factors. Some individuals with achondroplasia require multiple procedures and surgeries to address specific functional or anatomical concerns. About Ascendis Pharma A/SAscendis Pharma is a global biopharmaceutical company focused on applying our innovative TransCon technology platform to make a meaningful difference for patients. Guided by our core values of Patients, Science, and Passion, and following our algorithm for product innovation, we apply TransCon to develop new therapies that demonstrate best-in-class potential to address unmet medical needs. Ascendis is headquartered in Copenhagen, Denmark, and has additional facilities in Europe and the United States. Please visit ascendispharma.com to learn more. Forward-Looking Statements This press release contains forward-looking statements that involve substantial risks and uncertainties. All statements, other than statements of historical facts, included in this press release regarding Ascendis’ future operations, plans and objectives of management are forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. Examples of such statements include, but are not limited to, statements relating to (i) planned regulatory filings for infants 0 to <2 years of age with achondroplasia, (ii) the anticipated timing of a regulatory decision for the Marketing Authorisation Application for TransCon CNP as a monotherapy for children with achondroplasia, (iii) the potential benefits of TransCon CNP monotherapy and combination therapy with TransCon hGH, (iv) Ascendis’ clinical development activities, including the teACH, reACHin, and AttaCH trials, (v) Ascendis’ commercialization activities and efforts to provide access to YUVIWEL, and (vi) Ascendis’ ability to apply its TransCon technology platform to develop new therapies with best-in-class potential. Ascendis may not actually achieve the plans, carry out the intentions or meet the expectations or projections disclosed in the forward-looking statements and you should not place undue reliance on these forward-looking statements. Actual results or events could differ materially from the plans, intentions, expectations and projections disclosed in the forward-looking statements. Various important factors could cause actual results or events to differ materially from the forward-looking statements that Ascendis makes, including, without limitation: dependence on third‑party manufacturers, distributors, and service providers for Ascendis’ products and product candidates; risks related to regulatory review and approval, including the possibility of delays, requests for additional data or analyses, restrictions or limitations on use, approval with labeling that is more limited than expected, or failure to obtain approval in the United States, European Union, or other jurisdictions; clinical development risks, including that results from ongoing or future trials may not confirm earlier data; unforeseen safety or efficacy findings in development programs or on‑market products; manufacturing, supply chain, quality, or logistics issues that could delay development or commercialization; unforeseen expenses related to commercialization of any approved Ascendis products; unforeseen research and development or selling, general and administrative expenses and other costs impacting Ascendis’ business generally; market acceptance, pricing, and reimbursement challenges, including payer coverage decisions and health technology assessments; competitive developments, including new or improved therapies; intellectual property protection, freedom‑to‑operate, and litigation risks; Ascendis’ ability to obtain additional funding, if needed, to support its business activities; cybersecurity, data privacy, and information technology disruptions; and the impact of international economic, political, legal, compliance, public health, and business factors, including tariffs, trade policies, currency fluctuations, and geopolitical events. For a further description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as risks relating to Ascendis’ business in general, see Ascendis’ Annual Report on Form 20-F filed with the U.S. Securities and Exchange Commission (SEC) on February 11, 2026, and Ascendis’ other future reports filed with, or submitted to, the SEC. Forward-looking statements do not reflect the potential impact of any future licensing, collaborations, acquisitions, mergers, dispositions, joint ventures, or investments that Ascendis may enter into or make. Ascendis does not assume any obligation to update any forward-looking statements, except as required by law. Ascendis, Ascendis Pharma, the Ascendis Pharma logo, the company logo, TransCon, SKYTROFA® and YUVIWEL® are trademarks owned by the Ascendis Pharma group. © August 2026 Ascendis Pharma A/S. Investor Contact:Media Contact:Chad FugereMelinda BakerAscendis PharmaAscendis Pharma+1 (650) 519-7494+1 (650) 709-8875

2026-07-29

·QQ浏览器

2026 上半年 FDA 新药盘点：PROTAC、ADC、长效胰岛素等创新分子落地临床_ MedChemExpress

截至 2026 年 6 月，FDA 已批准多款创新药上市，覆盖肿瘤、代谢疾病、罕见病等多个热门领域。本文将带您一起梳理 2026 年上半年 FDA 新药批准情况，快速把握当下药物研发的关键方向。Copper histidinate (Zycubo)原研公司：Sentynl Therapeutics, Inc. & Cyprium Therapeutics, Inc.批准日期：1/12/2026Copper histidinate 是一种生物利用度高的铜替代疗法，用于治疗门克斯病。门克斯病是一种由铜转运蛋白基因 ATP7A 突变引起的遗传性疾病。由于门克斯病患者无法从消化道吸收铜，因此需通过每日皮下注射的方式给药。欧盟委员会已授予 Copper histidinate 针对门克斯病的孤儿药设计资格[1]。图 1 . Copper histidinate 在门克斯病治疗中的关键进展里程碑[1]。Difamilast(Adquey)原研公司：Otsuka Pharmaceuticals批准日期：2/12/2026特应性皮炎 (Atopic dermatitis, AD) 是一种常见的慢性复发性炎症性皮肤病。Difamilast 是一种磷酸二酯酶 4 (PDE 4) 抑制剂，该药物具有抗瘙痒和抗炎作用，起效迅速。II 期和 III 期临床试验表明，Difamilast 对成人及儿童患者均有效且耐受良好。早在 2021 年 Difamilast 成为第一个在日本获得生产与上市许可的用于治疗成人及儿童 (2 岁及以上) AD 的 PDE 抑制剂[2]。Milsaperidone(Bysanti)原研公司：Vanda Pharmaceuticals批准日期：2/20/2026Milsaperidone 是一种非典型抗精神病药物，用于治疗精神分裂症、双相 I 型障碍和重度抑郁症。Milsaperidone 是 iloperidone(Fanapt®) 的活性代谢产物，iloperidone 自 2009 年起在美国获批用于治疗精神分裂症，并于 2024 年起用于双相 I 型障碍的急性期治疗。临床试验表明 Milsaperidone 与 iloperidone 在药代动力学方面具有生物等效性，可用于成人精神分裂症的治疗，以及双相 I 型障碍伴发躁狂或混合发作的急性期治疗[3]。Pegzilarginase-nbln (Loargys)原研公司：Immedica Pharma批准日期：2/23/2026精氨酸酶 1 缺乏症 (ARG1-D) 是一种极为罕见的尿素循环障碍，其特征为高精氨酸血症以及进行性神经系统功能障碍，包括肌张力增高、运动功能丧失和生活质量下降。传统的饮食蛋白限制和氨清除剂治疗通常难以实现充分的代谢控制或防止神经系统恶化。Pegzilarginase (重组人精氨酸酶 1) 是第一个针对 ARG1-D 的疾病修饰疗法[4]。Navepegritide (Yuviwel)原研公司：Ascendis Pharma批准日期：2/27/2026软骨发育不全症是最常见的骨骼发育异常，是一种由 FGFR3 基因的激活突变引起的遗传性疾病，导致长骨的软骨内骨化障碍。临床表现包括身材比例失调和多系统并发症。目前的治疗手段多为支持性护理、手术延长肢体长度以及重组人生长激素，后两者分别存在较大风险且效果有限。近年来，靶向分子疗法已作为有前景的替代方案出现。Navepegritide 是一种长效 C 型钠尿肽 (C-type natriuretic peptide, CNP) 前体药物，可持续维持 MAPK 抑制作用[5]。图 2 . 与安慰剂相比，Navepegritide 治疗在第 52 周时的年化生长速度显著更高[5]。Linerixibat(Lynavoy)原研公司：GSK批准日期：3/17/2026Linerixibat 是一种口服的肠胆汁酸转运蛋白 (IBAT) 抑制剂，用于治疗原发性胆汁性胆管炎 (PBC) 患者严重的瘙痒症状[6]。PBC 是一种慢性自身免疫性肝内胆汁淤积性疾病伴随着疲劳和瘙痒的症状。Linerixibat 通过抑制 IBAT，减少胆汁酸在末端回肠的重吸收，打断肠肝循环，从而降低胆汁淤积性瘙痒相关的胆汁酸及其他介质水平[6]。图 3 . Linerixibat 通过阻断胆汁酸在 PBC 患者的再吸收，减轻胆汁淤积性瘙痒[7]。Icotrokinra(Icotyde)原研公司：Johnson & Johnson批准日期：3/17/2026银屑病是一种慢性、免疫介导的炎症性疾病，对生活质量有显著影响。Icotrokinra 是一种口服大环肽药物，是第一个口服的 IL-23R 拮抗剂，能够实现高水平且持久的皮肤清除效果，具有良好的安全性，在兼顾选择性、口服与 IL-23R 阻断的同时具有最小的副作用，并具有和药物相互作用的潜力[8]。图 4. Icotrokinra 选择性阻断 IL-23 信号通路，阻止 IL-23 异二聚体 (p19/p40) 与 IL-23R/IL-12Rβ1 受体复合物在 IL-23R⁺ 细胞上的结合，从而抑制下游依赖 IL-23 的炎症信号传导[8]。Tividenofusp alfa-eknm(Avlayah)原研公司：Denali Therapeutics批准日期：3/24/2026Tividenofusp alfa 是一种由唾液酸-2-硫酸酶 (iduronate-2-sulfatase) 与工程化转铁蛋白受体结合的 Fc 结构域融合而成的药物，用于治疗黏多糖贮积症 II 型 (MPS II) 的神经系统和外周系统表现。MPS II 是一种罕见的溶酶体疾病，可导致多系统进行性退化及神经系统功能下降[9]。Relacorilant(Lifyorli)原研公司：Corcept Therapeutics批准日期：3/25/2026Relacorilant 是一种首创的特异性糖皮质激素受体拮抗剂，通过减少皮质醇信号传导来提高肿瘤对化疗的敏感性。目前，Relacorilant 被批准联合白蛋白紫杉醇 (nab-paclitaxel)，治疗接受过 1-3 线全身治疗的成人铂耐药性上皮性卵巢癌、输卵管癌或原发性腹膜癌[10]。图 5 . Relacorilant 对诱导紫杉醇所致肿瘤细胞凋亡的信号通路的影响[10]。Insulin icodec-abae(Awiqli)原研公司：Novo Nordisk批准日期：3/26/2026Insulin icodec 是一种新型每周一次的基础胰岛素类似物，已获批用于改善 2 型糖尿病成人患者的血糖控制。其作用机制涉及可逆性与白蛋白结合，形成循环沉积库，使半衰期延长至约一周，并提供稳定的降糖效果。多项随机、治疗目标导向的临床试验证据表明，每周一次的 Insulin icodec 在血糖控制方面与每日一次的基础胰岛素类似物 (如甘精胰岛素和地特胰岛素) 相当，在某些情况下甚至更优。由于注射频率降低，该药物在提高治疗便利性和依从性方面具有显著优势[11]。Orforglipron(Foundayo)原研公司：Eli Lilly and Company批准日期：4/1/2026Orforglipron 是一种口服小分子胰高血糖素样肽-1 受体激动剂 (GLP-1 RA)，在 2 型糖尿病 (T2DM) 患者及非糖尿病成人中均显示出显著的减重和降糖益处。该药物可在全天任何时间服用，对饮食和饮水不加以限制。Orforglipron 在血糖控制方面优于 Dapagliflozin，其耐受性特征与 GLP-1 受体激动剂类药物一致[12]。Doravirine and Islatravir(Idvynso)原研公司：MSD批准日期：4/20/2026Doravirine 和 islatravir 是一种每日一次的单片剂方案，包含两种作用机制互补且耐药谱不同的强效抗逆转录病毒药物。Doravirine 是一种新一代非核苷类逆转录酶抑制剂，用于治疗 HIV 感染。在美国、中国、非洲及其他地区，该药以固定剂量复方片剂形式与核苷逆转录酶抑制剂联合使用。Islatravir 是一种第一代核苷逆转录酶转运抑制剂，在外周血单核细胞 (PBMCs) 中具有高活性和长半衰期。目前 Doravirine 和 islatravir 以双药单片方案，用于治疗成人 HIV-1 感染[13]。Vepdegestrant(Veppanu)原研公司：Pfizer批准日期：5/1/2026Vepdegestrant 是全球第一款获批的 PROTAC 药物，是一种口服的、PROTAC 雌激素受体 (ER) 降解剂。用于治疗 ER 阳性、HER2 阴性、ESR1 突变的晚期或转移性乳腺癌成人患者，且患者在接受至少一线内分泌治疗后出现疾病进展[14]。图 6. Vepdegestrant 的化学结构[14]。Sonrotoclax(Beqalzi)原研公司：BeOne Medicines批准日期：5/13/2026Sonrotoclax 是第一个获批用于复发或难治性滤泡细胞淋巴瘤的 BCL-2 抑制剂，该药物与 BCL-2 结合的能力比 Venetoclax 高出十倍以上，且具有更清洁的血小板毒性特征。此外，Sonrotoclax 能有效抑制 Venetoclax 耐药的 BCL2 突变体，例如 G101V[15]。目前 Sonrotoclax 批准用于治疗既往接受过至少两项系统治疗、且其中包括 BTK 抑制剂的复发或难治性 (R/R) 套细胞淋巴瘤 (MCL) 成人患者。图 7 . Sonrotoclax 是一种第二代 BCL2 抑制剂，对多种 BCL2 突变体具有较强的结合亲和力[15]。Baxdrostat(Baxfendy)原研公司：AstraZeneca批准日期：5/15/2026醛固酮水平异常升高是无法控制的高血压 (uHTN) 和难治性高血压 (rHTN) 的常见特征，且在血压升高之外，仍是导致不良心肾结局的主要病理生理因素。Baxdrostat 是一种选择性醛固酮合成酶抑制剂，在针对难治性高血压患者开展的第 2 期临床试验中，已证实其可剂量依赖性地降低静坐收缩压 (SBP)[16]。图 8 . Baxdrostat 用于控制不佳和难治性高血压的相关信息[16]。Bulevirtide-gmod(Hepcludex)原研公司：Gilead Sciences批准日期：5/22/2026Bulevirtide 是第一个能够失活 HBV 和 HDV 受体、与 HBV 竞争 Sodium-taurocholate co-transporting polypeptide (SCTP) 的药物。SCTP 是为 HBV 和 HDV 的真正受体，可阻断肝细胞中的 HBV 感染，并参与 HBV 转录抑制过程[17]。实验结果表明，经过 48 周的 Bulevirtide 治疗后，慢性丁型肝炎患者的 HDV RNA 和丙氨酸氨基转移酶 (ALT) 水平均有所下降。图 9 . Bulevirtide 的抗病毒机制[17]。Pivekimab sunirine-pvzy (Decnupaz)原研公司：AbbVie批准日期：5/27/2026Pivekimab sunirine 是一种突破性的抗体-药物偶联物 (ADC)，包含高亲和力的 CD123 抗体、可裂解连接臂以及新型吲哚啉苯并咪唑类伪二聚体治疗组分。CD123 在多种血液系统恶性肿瘤中过度表达，包括急性髓系白血病。目前批准用于治疗母细胞性浆细胞样树突状细胞肿瘤 (BPDCN) 成人患者，是第一个获批用于治疗 BPDCN 的 ADC 药物，第一个获批的 CD123 ADC。Cefepime and zidebactam(Zaynich)原研公司：Wockhardt批准日期：5/29/2026Cefepime 是四代头孢，对需氧性革兰氏阳性菌和阴性菌具有广谱抗菌活性，包括铜绿假单胞菌。目前批准治疗中重度肺炎、复杂性和非复杂性尿路感染、复杂性腹腔内感染以及非复杂性皮肤和皮下组织感染，同时还用于发热性中性粒细胞减少症患者的经验性治疗。Zidebactam 是一种 β-内酰胺酶增强型抗生素，具有双重作用机制，包括与革兰氏阴性菌的 PBP2 结合以及抑制 β-内酰胺酶。Cefepime 和 zidebactam，用于治疗包括肾盂肾炎在内，由敏感的革兰氏阴性病原体引起的成人复杂尿路感染 (cUTI)[18]。Ensitrelvir (Xocova)原研公司：Shionogi批准日期：5/29/2026Ensitrelvir 是一款口服 SARS-CoV-2 主要蛋白酶抑制剂，用于治疗 SARS-CoV-2 感染。它是第一个单体、非肽类、非共价、小分子抗病毒药物。在新冠患者症状出现后 72 小时内，对接触者使用 Ensitrelvir 可有效预防其感染新冠病毒[19]。Cipeofol(Cypsedo)原研公司：海思科批准日期：5/29/2026Cipepofol 是丙泊酚 (Propofol) 的类似物，其药效约为丙泊酚的 4-5 倍，且具有良好的安全性，目前被批准应用于诱导接受手术的成人全身麻醉。Cipepofol 于 2019 年在中国获批用于临床。Cipepofol 是一种 GABAA 受体的直接激动剂和正变构调节剂。Cipepofol 可激活 Sirtuin1 (Sirt1)/Nrf2 通路。Cipepofol 通过减少心脏氧化应激、炎症反应和心肌细胞凋亡来保护心脏免受 ‌Isoproterenol‌诱导的心肌梗死[20]。Gadoquatrane (Ambelvist)原研公司：Bayer批准日期：6/12/2026Gadoquatrane 是一种四聚体、大环结构的细胞外钆基 MRI 对比剂，具有高弛豫率和稳定性。在人体血浆中，60% 的钆浓度下，Gadoquatrane 和 Gadobutrol 的结果几乎完全重叠。这意味着在临床应用中，在更低的体重的剂量下，Gadoquatrane 产生的信号增强效果与 Gadobutrol 基本相当[21]。Tebpenem pivoxil (Utebzi)原研公司：GSK批准日期：6/17/2026Tebipenem pivoxil 是一种口服的碳青霉烯类抗生素，对多种耐药菌株具有活性，被批用于治疗成人复杂性尿路感染 (cUTI)，包括肾盂肾炎等肾脏炎症的药物。Tebipenem pivoxi 是美国第一个获批的口服碳青霉烯类抗生素[22]。Veligrotug-vvze (Lumvoa)原研公司：Viridian Therapeutics批准日期：6/26/2026甲状腺眼病 (TED) 是格雷夫斯病最常见的甲状旁腺外表现，每年每 10,000 人中约有 1.9 例。其发病机制涉及由 TSH 受体抗体 (TRAbs) 介导的自身免疫反应，导致炎症、成纤维细胞活化以及眶部组织重塑[23]。IGF-1R 拮抗剂可减轻 TED 引起的炎症和突眼。Veligrotug 是一种选择性的、完全的 IGF-1R 拮抗剂和抗体，对人 IGF-1R 蛋白表现出高亲和力结合 (KD=0.55 nM)，并在 HOCF 细胞中内源性表达的 IGF-1R 上表现出良好结合能力（平均 EC50=2.41 nM）[24]。图 10. IGF-1R抗体 (Veligrotug) 的独特特征[24]参考文献[1] Blair HA. Copper Histidinate: Pediatric First Approval. Paediatr Drugs. 2026 Apr 22.[2] Freitas E, Torres T. Difamilast for the treatment of atopic dermatitis. J Int Med Res. 2023 Jun;51(6):3000605231169445.[3] Shirley M. Milsaperidone: First Approval. Clin Drug Investig. 2026 May 29.[4] Faraguna MC, et al. Pegzilarginase in Arginase 1 Deficiency: Clinical and Biochemical Effects of Treatment Initiation, Discontinuation and Re-Initiation. Children (Basel).[5] Savarirayan R, et al. Once-Weekly Navepegritide in Children With Achondroplasia: The APPROACH Randomized Clinical Trial. JAMA Pediatr.[6] Hirschfield GM, et al. Linerixibat in patients with primary biliary cholangitis and cholestatic pruritus (GLISTEN): a randomised, multicentre, double-blind, placebo-controlled, phase 3 trial. Lancet Gastroenterol Hepatol. 2026 Jan;11(1):22-33.[7] Levy C, et al. GLIMMER: A Randomized Phase 2b Dose-Ranging Trial of Linerixibat in Primary Biliary Cholangitis Patients With Pruritus. Clin Gastroenterol Hepatol. 2023 Jul;21(7):1902-1912.e13.[8] Ferreira C, Torres T. Icotrokinra: An Oral Interleukin-23 Receptor Antagonist Peptide for the Treatment of Psoriasis. Am J Clin Dermatol. 2026 May;27(3):479-492.[9] Muenzer J, et al. An Intravenous Brain-Penetrant Enzyme Therapy for Mucopolysaccharidosis II. N Engl J Med. 2026 Jan 1;394(1):39-50.[10] Colombo N, et al. Relacorilant + Nab-Paclitaxel in Patients With Recurrent, Platinum-Resistant Ovarian Cancer: A Three-Arm, Randomized, Controlled, Open-Label Phase II Study. J Clin Oncol. 2023 Oct 20;41(30):4779-4789.[11] Abid MA, et al. A paradigm shift in diabetes care: clinical implications of once-weekly insulin icodec. Ann Med Surg (Lond). 2026 May 12;88(6):3787-3790.[12] Welch M, et al. Orforglipron compared with dapagliflozin in adults with type 2 diabetes and inadequate glycaemic control with metformin (ACHIEVE-2): a multicentre, randomised, non-inferiority, open-label, phase 3 trial. Lancet. 2026 Jun 8:S0140-6736(26)00800-7.[13] Daly MB, et al. Oral Islatravir in Macaques Decreases Lymphocytes and Monocytes and Is Associated with Immune Alterations. Pharmaceutics. 2026 Mar 20;18(3):381.[14] Tang C, et al. Insights Into Vepdegestrant (ARV-471): The First-in-Class Estrogen Receptor Proteolysis-Targeting Chimera Approaching Food and Drug Administration Approval for Breast Cancer. ChemMedChem. 2026 Mar 27;21(6):e202501111.[15] Liu J, et al. Sonrotoclax overcomes BCL2 G101V mutation-induced venetoclax resistance in preclinical models of hematologic malignancy. Blood. 2024 May 2;143(18):1825-1836.[16] Flack JM, et al. Baxdrostat for uncontrolled and resistant hypertension: rationale and design of the Phase 3 clinical trials BaxHTN, BaxAsia, and Bax24. Hypertens Res. 2025 Nov;48(11):2911-2923.[17] Cheng D, et al. Clinical effects of NTCP-inhibitor myrcludex B. J Viral Hepat. 2021 Jun;28(6):852-858.[18] Lepak AJ, et al. WCK 5222 (Cefepime/Zidebactam) Pharmacodynamic Target Analysis against Metallo-β-lactamase producing Enterobacteriaceae in the Neutropenic Mouse Pneumonia Model. Antimicrob Agents Chemother. 2019 Sep 9;63(12):e01648-19.[19] Hayden FG, et al. Ensitrelvir for Covid-19 Postexposure Prophylaxis in Household Contacts. N Engl J Med. 2026 May 14;394(19):1905-1915.[20] Yang Y, et al. Ciprofol attenuates the isoproterenol-induced oxidative damage, inflammatory response and cardiomyocyte apoptosis. Front Pharmacol. 2022 Nov 22;13:1037151.[21] Jost G, et al. Signal Enhancement of Gadoquatrane for Standard Clinical Pulse Sequences: A Phantom Study in Human Plasma. Invest Radiol. 2026 Apr 2.[22] Nuzhat S, et al. Tebipenem pivoxil as an alternative to ceftriaxone for clinically non-responding children with shigellosis: a randomised non-inferiority trial protocol. BMJ Open. 2025 Feb 25;15(2):e088449.[23] Niño Alarcón A, et al. Reactivation after total thyroidectomy? A thyroid eye disease case that challenges established clinical paradigms. Endocrinol Diabetes Metab Case Rep. 2026 Jun 9;2026(2):EDM250173.[24] Kaplan R, et al. Preclinical pharmacology, pharmacokinetics, and pharmacodynamics of veligrotug, a full antagonist antibody to the IGF-1 receptor in development for thyroid eye disease. MAbs. 2025 Dec;17(1):2585616.

100 项与那韦培肽相关的药物交易

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适应症	国家/地区	公司	日期
软骨发育不全	美国	Ascendis Pharma A/S	2026-02-27
软骨发育不全	美国	Ascendis Pharma Growth Disorders A/S	2026-02-27

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研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


NCT05598320 (ApproaCH \| ACcomplisH, CTgov)	临床2/3期	软骨发育不全	84	Navepegritide (Navepegritide)	簾夢窪製餘衊廠窪鑰積(鹽夢襯壓鑰壓築襯衊壓) = 襯憲艱衊壓構簾艱鏇簾鏇糧繭壓鬱憲膚艱衊膚 (築遞鬱齋選製膚廠壓夢, 願範糧範憲衊淵齋淵艱 ~ 憲齋遞選憲獵襯鹹襯襯) 更多	-	2026-07-14
				Placebo for Navepegritide (TransCon CNP) (Placebo for Navepegritide)	簾夢窪製餘衊廠窪鑰積(鹽夢襯壓鑰壓築襯衊壓) = 襯構廠鑰築顧襯齋膚艱鏇糧繭壓鬱憲膚艱衊膚 (築遞鬱齋選製膚廠壓夢, 繭憲網鬱鹽願構餘遞膚 ~ 鑰繭鹽鏇築艱鑰艱衊築) 更多
NCT05598320 \| NCT06433557 (COACH, Pubmed \| Eur J Endocrinol)	临床2期	软骨发育不全	21	-	餘衊範製範築艱簾夢餘(簾鏇鑰餘鬱簾鏇遞齋餘) = 壓繭觸鬱夢蓋築製繭蓋蓋鑰網鏇築淵鹽顧憲鹽 (範夢繭膚築觸憲齋築艱 ) 更多	积极	2026-06-01
				-	餘衊範製範築艱簾夢餘(簾鏇鑰餘鬱簾鏇遞齋餘) = 遞製窪襯遞繭鹹鹽廠觸蓋鑰網鏇築淵鹽顧憲鹽 (範夢繭膚築觸憲齋築艱 ) 更多
NCT04085523 \| NCT05598320 (FDA_CDER) 人工标引	N/A	软骨发育不全一线	-	Navepegritide 0.1 mg/kg (Trial 1)	醖網襯鑰齋鹹鑰鑰糧襯(襯繭鹽網膚糧繭鏇選願) = 構繭鑰齋製鬱鏇夢製壓遞齋窪鏇餘衊衊齋構壓 (夢範鬱範夢選獵糧繭網 ) 更多	积极	2026-02-27
				Placebo (Trial 1)	醖網襯鑰齋鹹鑰鑰糧襯(襯繭鹽網膚糧繭鏇選願) = 簾遞餘艱顧膚範糧製夢遞齋窪鏇餘衊衊齋構壓 (夢範鬱範夢選獵糧繭網 ) 更多
ApproaCH (ENDO2025)	临床3期	软骨发育不全	84	Navepegritide 100 μg/kg/week	憲壓築廠鏇製艱網壓鹹(顧襯願積襯鏇構衊壓廠) = 選鬱憲鏇壓鑰廠鬱淵醖襯遞鑰獵膚積蓋齋觸範 (築襯衊蓋範願選蓋餘築 ) 更多	积极	2025-07-12
				Placebo	憲壓築廠鏇製艱網壓鹹(顧襯願積襯鏇構衊壓廠) = 獵鹽蓋範鑰顧遞衊簾膚襯遞鑰獵膚積蓋齋觸範 (築襯衊蓋範願選蓋餘築 ) 更多
NCT05598320 (ApproaCH, PipelineReview) 人工标引	临床2/3期	软骨发育不全	84	TransCon CNP (ages 2-11 years)	簾築製遞艱製構襯窪願(遞顧壓鑰積糧糧壓鑰簾) = 獵齋築憲鹽壓鏇衊構夢衊鹹齋製顧膚遞廠遞觸 (鹽鹹積衊遞鹹艱夢蓋餘 ) 更多	积极	2024-09-16
				Placebo (ages 2-11 years)	簾築製遞艱製構襯窪願(遞顧壓鑰積糧糧壓鑰簾) = 築網蓋願築齋淵顧鹹窪衊鹹齋製顧膚遞廠遞觸 (鹽鹹積衊遞鹹艱夢蓋餘 ) 更多
NCT04085523 (ACcomplisH, Company_Website) 人工标引	临床2期	软骨发育不全	57	TransCon CNP	鹹膚觸糧觸鹹襯遞膚鏇(願夢範選願鹹鹹壓膚壓) = patients treated for one year at the pivotal 100μg/kg/week dose demonstrated significant improvements in well-being and physical functioning compared to placebo. 構餘醖憲遞鑰簾壓蓋蓋 (廠醖鑰鹹糧艱夢顧鹹繭 ) 更多	积极	2024-06-24
				Placebo
NCT04085523 (ACcomplisH, CTgov)	临床2期	软骨发育不全	57	TransCon CNP (TransCon CNP 6 mcg)	遞壓蓋艱願鬱蓋糧構鏇(蓋鹽鑰糧糧憲憲繭糧構) = 餘窪衊鹹網壓襯鬱築範襯構窪鹹簾願顧積製糧 (淵鑰齋觸製選膚製夢廠, 選憲網網遞範遞餘築餘 ~ 築範壓壓鏇憲醖獵餘獵) 更多	-	2024-03-07
				TransCon CNP (TransCon CNP 20 mcg)	遞壓蓋艱願鬱蓋糧構鏇(蓋鹽鑰糧糧憲憲繭糧構) = 鹽齋網膚淵艱餘願製製襯構窪鹹簾願顧積製糧 (淵鑰齋觸製選膚製夢廠, 淵膚選積膚簾鏇鹹網觸 ~ 襯醖襯鹽範鏇餘窪顧糧) 更多
NCT04085523 \| NCT05598320 (ACcomplisH, GlobeNewswire) 人工标引	临床2期	软骨发育不全	-	TransCon CNP 100 µg/kg/week	獵鹽築範襯遞鹹憲觸願(淵選壓淵鏇繭願膚繭顧): P-Value = 0.002 更多	积极	2023-12-20
				placebo
NCT05246033 (ACcomplisH China, PRNewswire \| NEWS) 人工标引	临床2期	软骨发育不全	24	TransCon CNPTransCon CNP 50μg/kg/week	齋構簾願範獵壓範餘鏇(網鏇窪襯鑰鑰鏇鑰廠壓) = 鏇衊積醖淵鏇鏇廠鬱簾蓋廠獵蓋顧夢衊顧鑰膚 (鑰憲簾壓積醖獵簾積遞 ) 达到	积极	2023-11-16
				TransCon CNPTransCon CNP 100μg/kg/week	齋構簾願範獵壓範餘鏇(網鏇窪襯鑰鑰鏇鑰廠壓) = 觸築製憲網淵襯觸範鏇蓋廠獵蓋顧夢衊顧鑰膚 (鑰憲簾壓積醖獵簾積遞 ) 达到
NCT04085523 (ACcomplisH, Pubmed)	临床2期	软骨发育不全	42	TransCon CNP 100 μg CNP/kg/week	積鹹範鏇鏇鹹蓋艱膚構(製願願壓齋鑰積蓋艱簾) = 遞鏇鑰鏇膚製鹽鬱廠繭膚製蓋糧壓鹽鏇鹹醖鬱 (餘壓簾艱鑰獵衊齋顧淵, 4.74 ~ 6.11)	积极	2023-11-01
				Placebo	積鹹範鏇鏇鹹蓋艱膚構(製願願壓齋鑰積蓋艱簾) = 鑰顧選積鹽廠壓積範壓膚製蓋糧壓鹽鏇鹹醖鬱 (餘壓簾艱鑰獵衊齋顧淵, 3.75 ~ 4.94)