Dive Brief:

• Ascendis Pharma said Friday the Food and Drug Administration granted accelerated approval to a treatment it’s developed for the most common type of dwarfism, ending what had been a monopoly for rival drugmaker BioMarin Pharmaceutical. 
• The new drug, previously known as TransCon CNP and now Yuviwel, is a once-weekly injection cleared for children aged two and older with achondroplasia and open growth plates. The treatment will compete with Voxzogo, a daily shot sold by BioMarin. 
• The accelerated clearance was based on Yuiwel’s ability to help spur growth as measured by an evaluation known as “annualized growth velocity,” or AGV. Ascendis needs to confirm those benefits in testing to uphold its approval. The company hasn’t yet announced Yuviwel’s price, but expects to make the drug available through prescribing physicians in the second quarter. 

Dive Insight:

Achondroplasia affects one out of every 15,000 to 40,000 newborns and is the most common form of dwarfism. It occurs due to genetic mutations that disrupt the body’s ability to convert cartilage into bones during early development. 

In 2021, BioMarin’s Voxzogo became the first drug approved for the condition. It’s meant to help spur bone growth in people with achondroplasia and is now BioMarin’s top-selling drug. But the treatment landscape is now shifting, seeding doubt about Voxzogo’s future and causing BioMarin to dial back its revenue projections. 

Yuviwel was originally slated to receive an FDA decision November, but the verdict was pushed back after Ascendis submitted additional information on post-marketing requirements. While that delay caused some consternation among analysts and investors, Voxzogo ultimately received a similar label as Voxzogo. The approval “represents a win amid broader industry uncertainty around FDA timelines,” wrote Stifel analyst Alex Thompson.

A recent check with prescribing doctors suggested that 40% of patients currently on injections could switch to the more convenient Yuviwel, wrote Cantor Fitzgerald analyst Li Watsek in a separate note. Uptake might be modest in 2026, with Wall Street analysts currently projecting $41.5M in sales. The drug could see greater momentum in 2027 and beyond due to an approval in the European Union, which is slated for the fourth quarter, and potential label expansions across different patient populations, Watsek wrote.

But more competition is already looming. San Francisco-based BridgeBio Pharma recently revealed positive data for a pill it’s developing for achondroplasia. BridgeBio plans to meet with drug regulators later this year to discuss approval plans. 

During a call with investors Monday, Ascendis said it’ll announce Yuviwel’s price either this week or next, and that the figure will be at a “premium commensurate with the clinical benefit Yuviwel offers to patients,” Watsek wrote.