BioMarin Responds to BridgeBio’s Challenge with an sNDA for Achondroplasia: Game On!

Biotech giant BridgeBio’s attempt to monopolize the achondroplasia market was dealt a severe blow on Tuesday, when rival Biomarin announced that the FDA had accepted its sNDA to expand Voxzogo (vosoritide) to children younger than five years old. This groundbreaking move means that BridgeBio may not have the exclusive hold on the market that it was hoping for.

The regulatory body is aiming to take action before October 21st, so make sure to stay up-to-date with the latest developments!

BioMarin recently submitted a sNDA for Voxzogo, bolstered by the results of a randomized, double-blinded, and placebo-controlled Phase II trial. The trial demonstrated that Voxzogo is safe for use in children under five years of age, just as it is for those who are older.

Hank Fuchs, M.D., President of Worldwide Research and Development at BioMarin, has expressed his concern about the lack of approved therapies for achondroplasia in very young children. He emphasizes the significance of this issue and calls for more research and development to address the needs of children with this condition.

“This approval will open doors for children with achondroplasia who have not yet reached the end of their growth plates, providing them with potential access to treatments that could aid their growth.”

Voxzogo made history in November 2021, becoming the first ever FDA-approved treatment for children with achondroplasia. And in January, this groundbreaking therapy was further expanded by the European Medicines Agency to include children under two years of age. This is a significant step forward for pediatric care and a major milestone for Voxzogo.

New Competition

Voxzogo’s recent sNDA announcement has been hot on the heels of BridgeBio’s promising Phase II results for infigratinib, their achondroplasia hopeful. After a six-month follow-up, the candidate’s highest dose level revealed a significant increase in the average growth rate of children, with an impressive 3.03 cm per year boost from their baseline growth rate.

Two children experienced an unexpected 8.8-cm growth spurt in their annual growth rate, despite not having any data from the first six months of the year.

Salim Syed, senior biotechnology analyst at Mizuho Securities, has noted that the data produced by BridgeBio is “in a completely new tier” and could be a major triumph over BioMarin, whose Voxzogo recently gained FDA approval this past November after improving growth rate by 1.57 cm per year in Phase III. With BridgeBio’s data, it promises to be a breakthrough in biotechnology.

If the data from Phase III holds up, it could have a significant impact on the preference for BridgeBio’s scripts. We predict that this could result in a major shift in favor of the company.

BioBridgeBio’s convenient oral formulation could revolutionize treatment for certain conditions by eliminating the injection-site reactions associated with BioMarin’s Voxzogo. With this new drug, patients no longer have to endure the discomfort of injections and can instead enjoy the simplicity of taking a pill.

Monday saw a dramatic shift in the market, with BridgeBio’s stock surging an impressive 73%, while BioMarin’s dipped by over 5%. This shift in favor of BridgeBio, a California-based competitor of BioMarin, marked a significant change in the market’s sentiment.

The achondroplasia arena continues to expand with the development of Tyra Biosciences’ FGFR inhibitor TYRA-300 and Ascendis Pharma’s investigational prodrug of the C-type natriuretic peptide. In November 2022, Ascendis Pharma announced that the prodrug had met its primary endpoint in a Phase II trial, adding to the growing number of potential treatments for achondroplasia. With the continued progress being made in this area, it is an exciting time for those affected by the condition.

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