BioMarin’s drug shows significant growth gains in children in late-stage trial

By Siddhi Mahatole

May 20 (Reuters) – BioMarin Pharmaceutical said on Wednesday its treatment for a rare condition that leads to short stature helped boost growth in children, meeting the main goal of a late-stage study and sending its shares up nearly 5% in extended trading.

Here are some details:

• The drug, Voxzogo, significantly increased the annualized growth rate in patients with hypochondroplasia after 52 weeks compared with placebo, with treated patients growing 2.33 cm more, the company said.

• Hypochondroplasia is a rare genetic skeletal disorder that affects bone growth and leads to short-limbed dwarfism.

• Patients receiving the therapy saw significant improvement in standing height and arm span, a key secondary goal of the study.

• The 80-patient study evaluated the drug in children aged 3 to 17 years.

• There are currently no approved treatments for the disorder, BioMarin said.

• Jefferies analyst Andrew Tsai said strong late-stage data for BioMarin’s Voxzogo supports the underlying growth biology and could de-risk BridgeBio’s <BBIO.O> oral rival infigratinib, which expects a study readout in the second half of 2026.

• “Our $1 billion peak sales estimate in achondroplasia/hypochondroplasia could be conservative,” Tsai added.

• Voxzogo was already approved in 2021 in the U.S. for patients with achondroplasia, a form of dwarfism.

• BioMarin plans to submit a supplemental application to U.S. regulators in the third quarter of this year.

(Reporting by Siddhi Mahatole in Bengaluru; Editing by Diti Pujara)