Yuviwel (navepegritide) / Ascendis, Teijin, Specialised Therap - LARVOL DELTA

Navepegritide therapy in infants with achondroplasia aged less than 2 years: Sentinel data from the reACHin Trial (ESPE 2026) - No abstract available

Genetic Disorders

Dual Therapy With Navepegritide and Lonapegsomatropin Demonstrated Sustained Efficacy and Safety in Children With Achondroplasia at Week 78 of the COACH Trial (ASBMR 2026) - No abstract available

Clinical • Late-breaking abstract • Genetic Disorders

Navepegritide in children with achondroplasia. (PubMed, Transl Pediatr) - No abstract available

Journal • Genetic Disorders

A Dose Escalation Trial Evaluating Safety, Efficacy, and Pharmacokinetics of Multiple Subcutaneous Doses of TransCon CNP Administered Once Weekly in Children With Achondroplasia (clinicaltrials.gov) - P2 | N=24 | Completed | Sponsor: Visen Pharmaceuticals (Shanghai) Co., Ltd. | Active, not recruiting ➔ Completed

Trial completion • Genetic Disorders

Improved Growth and Physical Functioning in Children with Achondroplasia Treated with Navepegritide in the ApproaCH Trial Open-Label Extension (ASBMR 2026) - No abstract available

Clinical • Genetic Disorders

Improved Growth and Physical Functioning in Children with Achondroplasia Treated with Navepegritide in the ApproaCH Trial Open-Label Extension (ASBMR 2026) - No abstract available

Clinical • Genetic Disorders

Continued Improvements in Lower Extremity Alignment in Navepegritide-Treated Children With Achondroplasia: Week 104 Results from the ApproaCH Trial (ASBMR 2026) - No abstract available

Clinical • Genetic Disorders

Continued Improvements in Lower Extremity Alignment in Navepegritide-Treated Children With Achondroplasia: Week 104 Results from the ApproaCH Trial (ASBMR 2026) - No abstract available

Clinical • Genetic Disorders

Assessing signs and impacts of achondroplasia: psychometric evaluation of the Achondroplasia Child Experience Measures. (PubMed, J Patient Rep Outcomes) - P2 | "The ACEMs are reliable and valid measures of the signs and impacts of achondroplasia on children. Incorporation of these measures in clinical and research settings can aid in assessing new treatments and improve our understanding of the experience of those living with achondroplasia."

Journal • Genetic Disorders • Orthopedics

Achondroplasia management in the era of targeted therapies: a meta-analysis of C-type natriuretic peptide analogs. (PubMed, J Endocr Soc) - "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. 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."

Journal • Retrospective data • Review • Dermatology • Genetic Disorders • Immunology • Orthopedics • Urticaria • FGFR3

Ascendis Pharma A/S has completed the sale of its U.S. Rare Pediatric Disease Priority Review Voucher to an undisclosed buyer for $187.5 million in cash before transaction-related expenses (StockTitan.net) - "The Priority Review Voucher was granted by the U.S. Food and Drug Administration following approval of YUVIWEL (navepegritide) in February 2026."

Commercial • Genetic Disorders

Navepegritide Combined with Lonapegsomatropin for the Treatment of Achondroplasia: An Open-Label Phase 2 Trial (ENDO 2026) - P2/3 | "One year of once weekly navepegritide and lonapegsomatropin were well tolerated and augmented proportionate growth in children with ACH, suggesting that lonapegsomatropin, through a distinct mode of action, complements navepegritide's therapeutic effects in children with ACH."

Clinical • P2 data • Genetic Disorders • FGFR3

Navepegritide: First Approval. (PubMed, Drugs) - "Navepegritide recently received accelerated approval in the USA for increasing linear growth in paediatric patients aged ≥ 2 years with achondroplasia with open epiphyses. This article summarizes the milestones in the development of navepegritide leading to this first approval for achondroplasia."

Journal • Genetic Disorders • Pediatrics • FGFR3

Navepegritide Combined with Lonapegsomatropin for the Treatment of Children with Achondroplasia: 52-Week Results from the Phase 2 COACH Trial. (PubMed, Eur J Endocrinol) - P2/3 | "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."

Journal • P2 data • Genetic Disorders

teACH: A Clinical Trial to Evaluate Efficacy and Safety of Navepegritide in Adolescents (12 - 18 Years of Age) With Achondroplasia. (clinicaltrials.gov) - P2 | N=24 | Recruiting | Sponsor: Ascendis Pharma A/S | Trial completion date: Jan 2027 ➔ Sep 2027 | Trial primary completion date: Dec 2026 ➔ Aug 2027

Trial completion date • Trial primary completion date • Genetic Disorders

YUVIWELⓇ (navepegritide). (PubMed, Clin Ther) - No abstract available

Journal

Ascendis Pharma A/S…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. (GlobeNewswire) - "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."

Launch US • Orphan drug • Reimbursement • Genetic Disorders

Growth Outcomes and Safety of Navepegritide in Children with Achondroplasia: Results of the ApproaCH Trial Open Label Extension (ACMG 2026) - "During the OLE of the ApproaCH trial, children continuing treatment with once-weekly navepegritide maintained increased AGV through 104 weeks. Children who switched from placebo to navepegritide at Week 52 demonstrated increased AGV at Week 104, similar to children who were treated with navepegritide in the DB period. The improvements in achondroplasia-specific and CDC-based height Z-scores in the OLE were consistent with improvements observed with navepegritide in the DB period."

Clinical • Genetic Disorders • Hypotension • Orthopedics • FGFR3

A Clinical Trial to Evaluate Efficacy and Safety of TransCon CNP Compared With Placebo in Infants (0 to <2 Years of Age) With Achondroplasia (clinicaltrials.gov) - P2 | N=72 | Recruiting | Sponsor: Ascendis Pharma A/S | Trial completion date: Mar 2027 ➔ Dec 2028 | Trial primary completion date: Mar 2026 ➔ Dec 2027

Trial completion date • Trial primary completion date • Genetic Disorders

FDA Approves Once-Weekly YUVIWEL (navepegritide) for Children with Achondroplasia Aged 2 Years and Older (GlobeNewswire) - "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 FDA based its approval of YUVIWEL on their review of the clinical package for TransCon CNP submitted with the Company’s New Drug Application, which included safety and efficacy data from three randomized, double-blind, placebo-controlled clinical trials....Ascendis expects to make YUVIWEL available through prescribing physicians in the United States during the early part of the second quarter of 2026."

FDA approval • Launch US • Genetic Disorders

Lower Extremity Alignment Improvements Correlate with Physical Functioning in Children with Achondroplasia Treated with Navepegritide: 52-Week ApproaCH Trial Results (ACMG 2026) - "Navepegritide demonstrated superiority over placebo in AGV and was associated with reductions in TFA at Week 52 in the ApproaCH trial. The associations between the improvements in genu varum and ACEM-PF support that changes in quantitative lower extremity alignment with navepegritide treatment had a positive impact on physical functioning. These results highlight that navepegritide, administered once weekly, may offer benefits beyond promoting linear growth that translate into meaningful improvements in the lives of children with achondroplasia."

Clinical • Genetic Disorders • Orthopedics

ApproaCH: A Clinical Trial to Evaluate Efficacy and Safety of TransCon CNP Compared With Placebo in Children With Achondroplasia (clinicaltrials.gov) - P2/3 | N=84 | Completed | Sponsor: Ascendis Pharma Growth Disorders A/S | Active, not recruiting ➔ Completed

Trial completion • Genetic Disorders

Ascendis Announces Extension of FDA Review Period for TransCon CNP (navepegritide) for Children with Achondroplasia (GlobeNewswire) - "...In response to the FDA’s ongoing review of the New Drug Application (NDA) for TransCon CNP (navepegritide) for children with achondroplasia,...the FDA has extended the PDUFA target action date by three months to February 28, 2026."

PDUFA • Genetic Disorders

Once-Weekly Navepegritide in Children With Achondroplasia: The APPROACH Randomized Clinical Trial. (PubMed, JAMA Pediatr) - P2/3 | "Moreover, navepegritide demonstrated additional potential health benefits beyond growth. ClinicalTrials.gov Identifier: NCT05598320."

Clinical • Journal • Genetic Disorders • Hypotension • Musculoskeletal Diseases • Obstructive Sleep Apnea • Orthopedics • Respiratory Diseases • Sleep Apnea • Sleep Disorder • FGFR3

Ascendis Pharma A/S…has submitted a Marketing Authorisation Application to the European Medicines Agency for TransCon CNP (navepegritide), a treatment for children with achondroplasia (Investing.com) - "The application is based on data from three randomized, double-blind, placebo-controlled clinical trials and up to three years of open-label extension data, including results from the pivotal ApproaCH Trial....In the United States, TransCon CNP is under priority review by the Food and Drug Administration with a target date of November 30, 2025."

EMA filing • PDUFA • Genetic Disorders