UPDATE 1-BioMarin's drug shows significant growth gains in children in late-stage trial

BioMarin Pharmaceutical Inc.

BioMarin Pharmaceutical Inc.

BMRN

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Adds analyst comment in bullets 6,7

By Siddhi Mahatole

- BioMarin Pharmaceutical BMRN.O 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.