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

NCT06079398

/ Recruiting临床2期

A Phase 2, Multicenter, Double-Blind, Randomized, Placebo-controlled Trial, Evaluating Safety, Tolerability, and Efficacy of Subcutaneous Doses of TransCon CNP Administered Once Weekly for 52 Weeks in Infants (0 to <2 Years of Age) With Achondroplasia Followed by an Open Label Extension (OLE) Period

This trial is a Phase 2, multicenter, double-blind, randomized (ratio 2:1 TransCon CNP vs. placebo), placebo-controlled trial, designed to evaluate the safety, tolerability, and efficacy of 100 μg CNP/kg of Navepegritide (TransCon CNP) administered SC once-weekly for 52 weeks in infants with genetically verified heterozygous ACH, aged 0 to < 2 years at the time of randomization.

开始日期2024-01-23

申办/合作机构

Ascendis Pharma A/S

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项与那韦培肽相关的文献（医药）

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 ; Smith, Alden ; Hoernschemeyer, Daniel G. ; 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

2024-11-01·Expert Review of Endocrinology & Metabolism

Progress in managing children with achondroplasia

Review

作者: Savarirayan, Ravi ; Taylor-Miller, Tashunka

INTRODUCTION:

Achondroplasia is a heritable disorder of the skeleton that affects approximately 300,000 individuals worldwide. Until recently, treatment for this condition has been purely symptomatic. Efficacious treatment options for children are now approved or are in clinical trials.

AREAS COVERED:

This review discusses key advances in the therapeutic management of children with achondroplasia, including vosoritide, the first approved drug, and other emerging precision therapies. These include navepegritide, a long-acting form of C-type natriuretic peptide, and infigratinib, a tyrosine kinase receptor inhibitor, summarizing trial outcomes to date.

EXPERT OPINION:

The advent of the first approved precision therapy for achondroplasia in vosoritide has been a paradigm shifting advance for children affected by this condition. In addition to changing their natural growth history, it is hoped that it will decrease their medical complications and enhance functionality. These new treatment options highlight the importance of prompt prenatal identification and subsequent testing of a suspected fetus with achondroplasia and counseling of families. It is hoped that, in the near future, families will have the option to consider a range of effective targeted therapies that best suit their child with achondroplasia, starting from birth should they choose.

2023-11-01·EClinicalMedicine

Once-weekly TransCon CNP (navepegritide) in children with achondroplasia (ACcomplisH): a phase 2, multicentre, randomised, double-blind, placebo-controlled, dose-escalation trial

Article

作者: Ma, Nina S ; Zarate, Yuri A ; Savarirayan, Ravi ; Mao, Meng ; Winding, Bent ; Sousa, Sérgio B ; McDonnell, Ciara ; Bober, Michael B ; Hoernschemeyer, Daniel G ; Bacino, Carlos A ; Legare, Janet M ; Smith, Alden ; Volck, Birgitte ; Schnabel, Dirk ; Chakraborty, Mukta ; Ljungberg, Merete ; Hofman, Paul L ; Giwa, Adebola ; White, Klane K ; Shu, Aimee D ; Högler, Wolfgang ; Quattrin, Teresa ; Abuzzahab, M Jennifer

Background:

TransCon CNP (navepegritide) is an investigational prodrug of C-type natriuretic peptide (CNP) designed to allow for continuous CNP exposure with once-weekly dosing. This 52-week phase 2 (ACcomplisH) trial assessed the safety and efficacy of TransCon CNP in children with achondroplasia.

Methods:

ACcomplisH is a global, randomised, double-blind, placebo-controlled, dose-escalation trial. Study participants were recruited between June 10, 2020, and September 24, 2021. Eligible participants were prepubertal, aged 2-10 years, with genetically confirmed achondroplasia, and randomised 3:1 to once-weekly subcutaneous injections of TransCon CNP (6, 20, 50, or 100 μg CNP/kg/week) or placebo for 52 weeks. Primary objectives were safety and annualised growth velocity (AGV). ACcomplisH is registered with ClinicalTrials.gov (NCT04085523) and Eudra (CT 2019-002754-22).

Findings:

Forty-two participants received TransCon CNP at doses of 6 μg (n = 10; 7 female), 20 μg (n = 11; 3 female), 50 μg (n = 10; 3 female), or 100 μg (n = 11; 6 female) CNP/kg/week, with 15 receiving placebo (5 female). Treatment-emergent adverse events (TEAEs) were mild or moderate with no grade 3/4 events reported. There were 2 serious TEAEs that were assessed as not related to TransCon CNP. Eleven injection site reactions occurred in 8 participants receiving TransCon CNP and no symptomatic hypotension occurred. TransCon CNP demonstrated a dose-dependent improvement in AGV. At 52 weeks, TransCon CNP 100 μg CNP/kg/week significantly improved AGV vs placebo (least squares mean [95% CI] 5.42 [4.74-6.11] vs 4.35 [3.75-4.94] cm/year; p = 0.0218), and improved achondroplasia-specific height SDS from baseline (least squares mean [95% CI] 0.22 [0.02-0·41] vs -0·08 [-0.25 to 0.10]; p = 0.0283). All participants completed the randomised period and continued in the ongoing open-label extension period receiving TransCon CNP 100 μg CNP/kg/week.

Interpretation:

This phase 2 trial suggests that TransCon CNP is effective, safe, with low injection site reaction frequency, and may provide a novel, once-weekly treatment option for children with achondroplasia. These results support TransCon CNP at 100 μg CNP/kg/week in the ongoing pivotal trial.

Funding:

Ascendis Pharma, A/S.

152

项与那韦培肽相关的新闻（医药）

2026-04-08

·BioSpace

New Data from Week 52 of the Ongoing COACH Trial Showed that TransCon® hGH Accelerated TransCon® CNP’s Benefits Beyond Linear Growth in Children with Achondroplasia

- Unprecedented improvements in arm span observed with TransCon CNP and TransCon hGH combination therapy, a measure highly meaningful to the achondroplasia community - Enhanced improvements in spinal canal dimensions observed with TransCon CNP and TransCon hGH combination therapy compared to TransCon CNP monotherapy - For the TransCon CNP treatment-naïve cohort, the improvement in tibial femoral angle (TFA) indicated enhanced straightening of the legs - Children treated with long-term TransCon CNP monotherapy maintained TFA in normal range in COACH COPENHAGEN, Denmark, April 08, 2026 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (Nasdaq: ASND) today announced new data demonstrating TransCon hGH accelerated TransCon CNP’s benefits beyond linear growth with substantial improvements in arm span, spinal canal dimensions, and lower limb alignment. The new data are from Week 52 of the ongoing Phase 2 COACH Trial of combination therapy with once-weekly TransCon CNP and once-weekly TransCon hGH in children with achondroplasia. Ascendis previously reported Week 52 COACH results that demonstrated mean annualized growth velocity exceeding the 97th-percentile of average stature children, without compromising safety or tolerability and with no acceleration of bone age. “It is encouraging to see data emerging beyond linear growth, including reported improvements in arm span, spinal dimensions, and leg alignment, which may help broaden how outcomes are understood,” said Michael Hughes, Chair of the Biotech Committee at the Little People of America. “Findings from the COACH Trial contribute to this expanding view and can be meaningful to consider as part of a more comprehensive understanding of treatment effects, alongside continued efforts to engage with the achondroplasia community. As research progresses, longer-term data will be important to understand how these outcomes may translate over time, including areas such as skeletal health, spinal complications, and the need for interventions.” New Data from Week 52 of the COACH Trial Unprecedented improvements in arm span observed at Week 52 with TransCon CNP and TransCon hGH combination therapy, a measure highly meaningful to the achondroplasia community, with the mean change from baseline in achondroplasia (ACH)-specific arm span Z-scores at Week 52 for TransCon CNP treatment-naïve and TransCon CNP-treated children in COACH were +1.02 and +0.66, respectively. The TransCon CNP treatment-naïve cohort improved +9.4 cm and the TransCon CNP-treated cohort improved +7.9 cm. By comparison, humeral gain by limb lengthening surgery is approximately 8 cm per arm and carries a high complication risk. 1 Mean of L1-L5 average changes in interpedicular distance (IPD) for TransCon CNP treatment-naïve and TransCon CNP-treated children on combination therapy in COACH were +1.7 mm and +1.1 mm, respectively, compared to +0.6 mm for children on TransCon CNP monotherapy in ApproaCH. Improvements in IPD offer the potential to reduce nerve compression and pain that can result from a narrowed spinal column. For the TransCon CNP treatment-naïve cohort, the mean change in tibial femoral angle (TFA) Z-score was -0.86 with combination therapy at Week 52 in COACH and was -0.47 for TransCon CNP monotherapy at Week 52 in ApproaCH, indicating enhanced straightening of the legs. Children previously treated with long-term TransCon CNP monotherapy for an average of 2.56 years maintained in normal range for TFA Z-score. For the TransCon CNP treatment-naïve cohort, the mean change in TFA was -3.0 degrees with combination therapy in COACH and was -1.3 degrees for children on TransCon CNP monotherapy at Week 52 in ApproaCH. Children previously treated with long-term TransCon CNP monotherapy for an average of 2.56 years maintained TFA treatment benefit in the setting of accelerated growth. All children completed 52 weeks of treatment and remain on therapy in COACH as of today. TransCon CNP (navepegritide) is a prodrug of C-type natriuretic peptide (CNP) administered once weekly, providing continuous exposure of active CNP to receptors on tissues throughout the body, including growth plates and skeletal muscle. TransCon hGH (lonapegsomatropin) is a prodrug of somatropin administered once weekly, providing sustained release of active, unmodified somatropin. TransCon CNP was approved under the trade name YUVIWEL ® by the U.S. Food & Drug Administration (FDA) in February 2026 and is under review by the European Medicines Agency as a monotherapy for children with achondroplasia. TransCon hGH is approved by the FDA, European Commission, and other regulatory agencies and marketed as SKYTROFA ® for the treatment of pediatric and adult growth hormone deficiency; it is investigational in achondroplasia and other indications. COACH Trial Design COACH 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 in children with achondroplasia aged 2 to 11 years. The trial included a cohort of TransCon CNP treatment-naïve children (N=12, mean age 4.67 years) and a cohort of previously TransCon CNP-treated children (N=9, mean age 7.89 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. “These unprecedented COACH Trial results demonstrate benefits beyond linear growth, reflecting meaningful clinical improvements identified by the achondroplasia community,” said Aimee D. Shu, M.D., Executive Vice President of Endocrine & Rare Disease Medical Science and Chief Medical Officer at Ascendis Pharma. “Across clinical trials, TransCon CNP has demonstrated unique benefits as monotherapy and now we see potential in combination with TransCon hGH to enhance these transformative changes for individuals with achondroplasia.” A slide presentation with these new COACH Trial Week 52 data can be found on the Investors & News section of the Ascendis Pharma website: . About Achondroplasia Achondroplasia 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/S Ascendis 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) the potential of TransCon CNP and TransCon hGH combination therapy as a differentiated therapy for short stature in the setting of growth hormone sufficiency, (ii) Ascendis’ ability to apply its TransCon technology platform to make a meaningful difference for patients, and (iii) Ascendis’ ability to apply TransCon to develop new therapies that demonstrate best-in-class potential to address unmet medical needs. 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, TransCon, SKYTROFA, and YUVIWEL are trademarks owned by the Ascendis Pharma group. © April 2026 Ascendis Pharma A/S. Investor Contacts: Media Contact: Chad Fugere Melinda Baker Ascendis Pharma Ascendis Pharma +1 (650) 519-7494 +1 (650) 709-8875 1 Hosny G, et al. International Orthopaedics . Published online March 16, 2026. doi:

临床结果临床2期上市批准

2026-04-07

·BioSpace

Ascendis Receives Orphan Drug Exclusivity and Launches YUVIWEL® (Navepegritide) in the United States

- YUVIWEL was granted orphan drug exclusivity by U.S. FDA to increase linear growth in pediatric patients 2 years of age and older with achondroplasia with open epiphyses - YUVIWEL is now commercially available in the U.S. with revenue now recognized following approval of first patients and initiation of therapy COPENHAGEN, Denmark, April 06, 2026 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (Nasdaq: ASND) today announced that YUVIWEL (navepegritide; developed as TransCon ® CNP) was granted orphan drug exclusivity by the U.S. Food & Drug Administration (FDA) and that YUVIWEL is now commercially available in the United States. YUVIWEL is the first and only once-weekly treatment approved by FDA to increase linear growth in children 2 years of age and older with achondroplasia with open epiphyses and is the only one to provide continuous systemic exposure to CNP over the weekly dosing interval. Multiple YUVIWEL prescriptions have already been approved for reimbursement for children in the U.S. enrolled through the Ascendis Signature Access Program ® (A.S.A.P.), with revenue now recognized following approval of first patients and initiation of therapy. Ascendis has established A.S.A.P. to provide support to children in the U.S. prescribed YUVIWEL and their caregivers throughout the treatment journey, including a dedicated Patient Access Liaison, programs designed to help navigate each step of the treatment process, and information about co-pay and other assistance programs for eligible patients. The program also provides resources for healthcare providers, including clinical education and assistance with prior authorization and appeals. Information about the A.S.A.P. program for YUVIWEL can be found at . “Our daughter has gained new independence and confidence with her growth, following treatment with YUVIWEL in a clinical trial,” said Brandi, mother of an 11-year-old child living with achondroplasia. “It is satisfying to know that this once-weekly treatment is now available to others in the achondroplasia community seeking pharmacological options to mitigate the potential impacts of this condition.” “We’re excited to see the FDA grant orphan drug exclusivity for YUVIWEL, acknowledging it as a highly differentiated treatment option,” said Jan Mikkelsen, President and Chief Executive Officer of Ascendis Pharma. “We have now delivered and commercialized our third FDA-approved TransCon product in a row, and remain on track to fulfill our objective of being the leader in Growth Disorders.” YUVIWEL (navepegritide) received Orphan Drug Designation from the U.S. FDA in February 2019. The FDA grants orphan designation to drugs that are intended for the safe and effective treatment, diagnosis, or prevention of rare diseases or disorders that affect fewer than 200,000 people in the United States, and that potentially may be safer or more effective than already approved products. Subject to certain exceptions, orphan designation provides a drug developer with certain benefits and incentives, including a seven-year period of U.S. marketing exclusivity upon approval of the product in the orphan-designated indication, waiver of FDA user fees, and tax credits for clinical research. The subsequent orphan drug exclusivity granted to YUVIWEL will run through February 27, 2033. YUVIWEL 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. It was approved by the FDA on February 27, 2026 under the agency’s Accelerated Approval Program. Continued approval for this indication, which was based on an improvement in annualized growth velocity (AGV), may be contingent upon verification and description of clinical benefit in confirmatory trial(s). The following information is intended for the U.S. audience only: USE AND IMPORTANT SAFETY INFORMATION What is YUVIWEL? YUVIWEL is a prescription medicine used to increase linear growth in children 2 years and older with achondroplasia with open growth plates (epiphyses). YUVIWEL is approved under accelerated approval based on improvement in annualized growth velocity. Continued approval may be contingent upon verification and description of clinical benefit in confirmatory trials. IMPORTANT SAFETY INFORMATION What are the possible side effects of YUVIWEL? YUVIWEL may cause serious side effects, including risk of low blood pressure. If your child experiences a decrease in blood pressure or symptoms of low blood pressure (dizziness, feeling tired, and/or nausea) while being treated with YUVIWEL, call your child’s healthcare provider. The most common side effects of YUVIWEL include injection site reactions (redness, itching, skin discoloration, bleeding, swelling, bruising, pain, and blistering). Before you give YUVIWEL to your child, tell the healthcare provider about all of your child’s medical conditions, including if they: have kidney problems are pregnant or plan to become pregnant. It is not known if YUVIWEL will harm the unborn baby are breastfeeding or plan to breastfeed. It is not known if YUVIWEL passes into breast milk Tell the healthcare provider about all the medicines your child takes , including prescription and over-the-counter medicines, vitamins, and herbal supplements. These are not all of the possible side effects of YUVIWEL. Call your doctor for medical advice about side effects. You are encouraged to report side effects to FDA at 1-(800) FDA-1088 or . You may also report side effects to Ascendis Pharma at 1-844-442-7236. Please see full Prescribing Information for YUVIWEL in the United States. About Achondroplasia Achondroplasia 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/S Ascendis 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) the potential for YUVIWEL to address the medical complications of achondroplasia in new ways, (ii) the anticipated timing of the subsequent orphan drug exclusivity granted to YUVIWEL, (iii) Ascendis’ ability to apply its TransCon technology platform to make a meaningful difference for patients, and (iv) Ascendis’ ability to apply TransCon to develop new therapies that demonstrate best-in-class potential to address unmet medical needs. 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, TransCon, and YUVIWEL ® are trademarks owned by the Ascendis Pharma group. © April 2026 Ascendis Pharma A/S. Investor Contacts: Media Contact: Chad Fugere Melinda Baker Ascendis Pharma Ascendis Pharma +1 (650) 519-7494 +1 (650) 709-8875

孤儿药上市批准

2026-04-03

·同花顺

2026年第一季度多肽疗法获多项监管批准与超亿美元融资，研发版图持续拓展

据药明康德（603259）消息，2026年第一季度，全球多肽疗法领域在监管审批、临床研究与产业合作方面均取得显著进展。以GLP-1类药物为代表的多肽疗法在肥胖、代谢疾病等领域持续突破，同时向肿瘤、自免疫疾病等更广泛适应症拓展。伴随研发热度，大型药企达成高额合作，创新公司也获得巨额资本支持，推动该领域快速发展。监管进展本季度，多款多肽药物在全球范围内获得重要监管批准或推荐：1. GLP-1类药物：诺和诺德高剂量司美格鲁肽（7.2mg）获美国FDA批准用于肥胖症；司美格鲁肽获欧洲EMA人用药品委员会推荐有条件批准用于代谢功能障碍相关脂肪性肝炎（MASH），有望成为首个在欧获批该适应症的GLP-1药物；其口服片剂获加拿大卫生部批准用于降低2型糖尿病患者心血管事件风险。2. 非GLP-1类药物：Ascendis Pharma的每周一次给药疗法Yuviwel获FDA加速批准，用于治疗软骨发育不全儿童。强生与Protagonist Therapeutics合作的口服多肽疗法Icotyde获FDA批准，用于中重度斑块状银屑病，是首个精准阻断IL-23受体的靶向口服多肽药物。Rhythm Pharmaceuticals的Imcivree扩展适应症获FDA批准，用于治疗获得性下丘脑性肥胖。此外，Sobi与Apellis Pharmaceuticals的Aspaveli获欧盟委员会批准，用于治疗C3肾小球病。临床进展临床研究方面，GLP-1相关疗法仍是焦点，其应用范围也逐步拓宽：1. 肥胖与代谢疾病：礼来的三重激素受体激动剂retatrutide在3期试验中显示可显著降低2型糖尿病患者体重与糖化血红蛋白。辉瑞的长效GLP-1受体激动剂PF-08653944在2b期试验中显示强劲减重效果，具备每月一次给药潜力。Viking Therapeutics的双重激动剂VK2735完成3期患者招募。Altimmune的pemvidutide获FDA突破性疗法认定用于MASH。诺和诺德的固定剂量复方CagriSema在3期试验中减重与改善血糖效果优于单用司美格鲁肽。2. 拓展至其他领域：礼来公布其GLP-1/GIP双重激动剂Zepbound与IL-17A单抗联合疗法，在银屑病及银屑病关节炎的3期试验中达到主要终点，提示GLP-1疗法与生物制剂联合的新策略。3. 其他疾病进展：Priavoid的口服多肽疗法PRI-002在2期试验中显示其淀粉样相关影像学异常发生率与安慰剂组相当。Parabilis Medicines的多肽疗法zolucatetide在1/2期试验中使一名家族性腺瘤性息肉病患者息肉数量与大小显著减少，硬纤维瘤直径缩小52.2%。研发合作与融资进展产业合作与融资活动活跃：1. 重大合作：阿斯利康与石药集团达成最高可达35亿美元的战略合作，共同推进8项针对肥胖与2型糖尿病的下一代疗法。诺华与Unnatural Products达成总额超17亿美元的大环肽合作。2. 融资支持：Parabilis Medicines完成超3亿美元F轮融资，用于推进zolucatetide开发。峰肽药业完成8900万美元B轮融资，以加速管线临床推进。一体化平台助力应对研发挑战随着多肽分子设计日益复杂（如脂肪酸修饰、PEG化、蛋白融合等），其对生物分析（如药代动力学研究与免疫原性评价）提出了更高要求。药明康德生物分析部构建了系统化分析解决方案，整合高灵敏度LC-MS/MS、免疫捕获-质谱等技术，以应对多肽药物在检测灵敏度、方法稳健性及免疫原性评价方面的特有挑战，支持创新多肽疗法从研究向临床开发加速迈进。原文：多项监管突破与上亿美元支持，多肽疗法在第一季度迎来多项进展 | Bilingual（来源：药明康德）关注同花顺财经（ths518），获取更多机会

突破性疗法临床结果临床3期加速审批临床2期

100 项与那韦培肽相关的药物交易

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研发状态

10 条最早获批的记录,

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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 Growth Disorders A/S	2026-02-27
软骨发育不全	美国	Ascendis Pharma A/S	2026-02-27

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临床结果

适应症

分期

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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


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)	鬱膚齋憲淵齋遞鑰糧餘(蓋衊壓鬱網築觸憲繭鏇) = 獵鏇鏇淵廠艱淵鹽製廠蓋糧鬱鹽鹹鹽壓鬱遞顧 (廠鏇蓋衊獵選膚構膚鹹 ) 更多	积极	2026-02-27
				Placebo (Trial 1)	鬱膚齋憲淵齋遞鑰糧餘(蓋衊壓鬱網築觸憲繭鏇) = 構憲艱鑰壓廠淵蓋夢蓋蓋糧鬱鹽鹹鹽壓鬱遞顧 (廠鏇蓋衊獵選膚構膚鹹 ) 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)	艱壓鏇窪襯觸構襯廠遞(憲鏇製獵願選積繭繭積) = 構簾襯廠齋糧鑰範獵夢築鹹觸膚範襯範憲製蓋 (糧獵顧淵廠醖簾製壓壓 ) 更多	积极	2024-09-16
				Placebo (ages 2-11 years)	艱壓鏇窪襯觸構襯廠遞(憲鏇製獵願選積繭繭積) = 齋醖壓鹽壓構艱膚壓廠築鹹觸膚範襯範憲製蓋 (糧獵顧淵廠醖簾製壓壓 ) 更多
NCT04085523 (ACcomplisH, Company_Website) 人工标引	临床2期	软骨发育不全	57	TransCon CNP	鏇膚夢壓夢齋齋衊鬱膚(範夢鹹選蓋獵獵鹹糧築) = patients treated for one year at the pivotal 100μg/kg/week dose demonstrated significant improvements in well-being and physical functioning compared to placebo. 窪築膚範選鹽齋齋範壓 (遞夢鏇鏇製製壓窪築願 ) 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)	選衊鑰網壓積積繭觸壓(窪膚窪廠築齋艱選廠壓) = 遞夢遞網選鹽網鏇構選遞積遞鬱範鹽襯窪製襯 (廠顧廠糧鬱鬱鏇艱鹹鬱, 願製廠糧繭築醖網窪選 ~ 醖鹽獵遞製糧積壓願淵) 更多	-	2024-03-07
				TransCon CNP (TransCon CNP 20 mcg)	選衊鑰網壓積積繭觸壓(窪膚窪廠築齋艱選廠壓) = 簾簾夢糧繭製窪蓋鹹選遞積遞鬱範鹽襯窪製襯 (廠顧廠糧鬱鬱鏇艱鹹鬱, 鹽淵淵襯鏇鹹衊鹹鹽鏇 ~ 壓鹹鑰艱齋衊積夢壓構) 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	廠鏇蓋築鏇鹹鹹遞築遞(遞範鬱鏇簾積廠憲鏇衊): 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	蓋範膚範鏇衊淵夢醖醖(醖選齋鏇鏇範製壓艱廠) = 獵糧觸憲獵顧衊鑰糧遞憲鑰壓窪選觸廠餘衊衊 (觸製衊憲廠衊齋願衊鑰 ) 达到	积极	2023-11-16
				TransCon CNPTransCon CNP 100μg/kg/week	蓋範膚範鏇衊淵夢醖醖(醖選齋鏇鏇範製壓艱廠) = 憲蓋齋醖構糧膚範淵糧憲鑰壓窪選觸廠餘衊衊 (觸製衊憲廠衊齋願衊鑰 ) 达到
NCT04085523 (ACcomplisH, Pubmed)	临床2期	软骨发育不全	42	TransCon CNP 100 μg CNP/kg/week	製淵窪廠餘鏇積膚壓製(鏇範淵鹹製觸簾衊壓遞) = 獵糧糧網鑰膚獵淵餘獵積構糧選鏇鹹窪鬱鬱構 (構窪顧糧艱膚淵製鏇艱, 4.74 ~ 6.11)	积极	2023-11-01
				Placebo	製淵窪廠餘鏇積膚壓製(鏇範淵鹹製觸簾衊壓遞) = 鬱鑰構窪廠鹹簾選範獵積構糧選鏇鹹窪鬱鬱構 (構窪顧糧艱膚淵製鏇艱, 3.75 ~ 4.94)
NCT05598320 (ACcomplisH, ENDO2023)	临床2期	软骨发育不全	57	TransCon CNP 6 μg/kg/week	衊製窪夢願壓壓築艱衊(餘窪遞餘遞淵範築糧衊) = 選鑰憲鹽壓壓糧構觸簾鬱構築遞繭膚鬱壓夢築 (製築餘選範積選餘鹹壓 )	积极	2023-10-05
				TransCon CNP 20 μg/kg/week	衊製窪夢願壓壓築艱衊(餘窪遞餘遞淵範築糧衊) = 願繭網鹹顧齋範憲鬱選鬱構築遞繭膚鬱壓夢築 (製築餘選範積選餘鹹壓 )