Ascendis Pharma (ASND) FDA announcement summary

Introduction and purpose

• FDA approved YUVIWEL (navepegritide) as the first once-weekly therapy for children aged 2 years and older with achondroplasia and open epiphyses, providing continuous systemic CNP exposure over the weekly dosing interval.

• Approval addresses unmet medical needs in achondroplasia, aiming to increase linear growth and improve quality of life, with a patient-focused mission and collaboration with advocacy groups.

• YUVIWEL is the third FDA-approved drug using TransCon technology.

• Approval was granted under the Accelerated Approval Program, based on improvement in annualized growth velocity (AGV).

• A Rare Pediatric Disease Priority Review Voucher was issued in connection with the approval.

Details of approval or decision

• YUVIWEL is indicated to increase linear growth in pediatric patients aged two years and older with open epiphyses, with no upper age limit.

• Approval is based on three randomized, double-blind, placebo-controlled trials, including the pivotal ApproaCH Trial, with continued approval contingent on confirmatory trials verifying clinical benefit.

• FDA approval was granted under the Accelerated Approval Program, based on improvement in AGV.

• YUVIWEL received a Rare Pediatric Disease Priority Review Voucher.

• YUVIWEL will be available through prescribing physicians and supported by patient services, including financial assistance.

Impact on industry and stakeholders

• YUVIWEL is the first and only once-weekly therapy for achondroplasia, expected to expand the therapeutic class and address significant unmet needs, with uptake from both treated and untreated pediatric patients.

• Over half of U.S. patients are concentrated in 100 skeletal dysplasia clinics, facilitating targeted launch.

• Commercial launch in the U.S. is planned for early Q2 2026, with international expansion and early access programs to follow.

• Patient support programs and financial assistance are in place to ensure broad access, with infrastructure and sales force scaled to leverage rare disease experience.

• Community engagement since 2017 shaped development and launch, with ongoing advocacy and support initiatives.