BioMarin answers BridgeBio’s challenge with sNDA for achondroplasia

Published: Mar 08, 2023

By Tristan Manalac


BioMarin refuses to give BridgeBio ground in their battle for the achondroplasia market. Tuesday, the rare disease biotech announced the FDA has accepted its supplemental New Drug Application (sNDA) to expand Voxzogo (vosoritide) to children younger than five years.

The regulatory body has a target action date of Oct. 21.

BioMarin backed its sNDA for Voxzogo with data from a randomized, double-blinded and placebo-controlled Phase II trial which showed the drug was just as safe in children under five years of age as it was in those who are older.

There are currently no approved therapies for achondroplasia in very young children, Hank Fuchs, M.D., president, Worldwide Research and Development, BioMarin, said in a statement.

“This approval could extend access to all children with achondroplasia whose growth plates are not closed,” Fuchs said.

Voxzogo notched its initial regulatory win in November 2021 and is the first FDA-approved treatment for children with achondroplasia. In January, the European Medicines Agency approved expanding Voxzogo’s indication to children under two years of age.

New Competition

Voxzogo’s sNDA comes a day after BridgeBio posted promising Phase II results for its own achondroplasia hopeful infigratinib. At the six-month follow-up, the candidate’s highest dose level increased the average growth rate of children by 3.03 cm per year, a significant improvement from their baseline growth rate.

Two children who had not yet had six months’ worth of data saw an 8.8-cm increase in their annual growth rate.

In a note to investors, Salim Syed, senior biotechnology analyst, Mizuho Securities, said BridgeBio’s data are “in a completely new tier” and could be a definite win over BioMarin, whose Voxzogo won the FDA’s approval in November 2021 after it improved growth rate by 1.57 cm per year in Phase III.

“If this data holds up in Phase III it would likely and massively drive script preference, we believe, in BridgeBio’s favor,” Syed said.

BridgeBio’s drug is an oral formulation, which is more convenient than and could do away with injection-site reactions associated with BioMarin’s Voxzogo.

The market also seems to favor BioMarin’s California-based competitor, with BridgeBio’s stock surging as much as 73% Monday, while BioMarin’s dipped by over 5%.

Also in the achondroplasia arena is Tyra Biosciences, which expanded the development of its FGFR inhibitor TYRA-300 into this indication and Ascendis Pharma, which in November 2022 revealed that its investigational prodrug of the C-type natriuretic peptide met its primary endpoint in a Phase II trial.

Source: BioSpace