Global pharma powerhouse Ascendis Pharma has announced that the US FDA has approved YUVIWEL (navepegritide), marking the first and only once-weekly therapy to increase linear growth in children aged 2 and older with achondroplasia. 

 

The treatment also delivers continuous systemic exposure to CNP over the weekly dosing interval. Continued approval will depend on verification of clinical benefit in confirmatory trials.

 

Achondroplasia, a rare genetic disorder, causes skeletal dysplasia and often increases the risk of muscular, neurological, and cardiorespiratory complications. YUVIWEL, a prodrug of C-type natriuretic peptide (CNP), is designed to provide ongoing exposure of active CNP to body tissues, counteracting the overactive FGFR3 signalling seen in achondroplasia.

 

“The approval of once-weekly YUVIWEL is a major step forward in the treatment of children with achondroplasia, giving physicians for the first time the option of prescribing a once-weekly medicine backed by compelling efficacy and excellent tolerability data from three randomized, double-blind, placebo-controlled clinical trials,” said Carlos A Bacino, Professor of Molecular and Human Genetics at Baylor College of Medicine and Texas Children’s Hospital.

 

The approval drew praise from advocacy groups. “Little People of America, the largest national advocacy and support organization for people with dwarfism, is committed to ensuring that the voices of people with dwarfism remain central in conversations about research and medical options such as YUVIWEL,” said the organization’s Board of Directors. 

 

“We champion dwarf and disability pride, advocate for inclusion and respect, and foster open dialogue across diverse perspectives. Our goal is to empower individuals and families to make healthcare decisions that reflect their own values and experiences, while pushing for research efforts and new treatment options such as this that could have the potential to support outcomes that truly matter to our community.”

 

The FDA based its decision on clinical data from three randomized, double-blind, placebo-controlled trials and up to three years of open-label extension data. The pivotal ApproaCH Trial results are published in JAMA Pediatrics.

 

“We are confident in YUVIWEL’s potential to transform the treatment of achondroplasia and are deeply grateful to patients, clinicians, and advocates for their many contributions to this important milestone,” said Jan Mikkelsen, President and CEO of Ascendis Pharma. 

 

“We have listened to advocacy groups for people with dwarfism to ensure we address what the community actually cares about. This reflects our ongoing commitment to pursue outcomes that patient communities have told us are important to them, and gives the achondroplasia community a new way to look at the promise of pharmacological treatment options.”

 

Ascendis plans to make YUVIWEL available through prescribing physicians in the U.S. in early Q2 2026, accompanied by patient support services and financial assistance through its Ascendis Signature Access Program.