UPDATE 1-BioMarin's drug shows significant growth gains in children in late-stage trial
BioMarin Pharmaceutical Inc. BMRN | 0.00 |
Adds analyst comment in bullets 6,7
By Siddhi Mahatole
May 20 (Reuters) - 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.
