BioMarin's drug shows significant growth gains in children in late-stage trial
BioMarin Pharmaceutical Inc. BMRN | 0.00 |
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 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 by the U.S. Food and Drug Administration or the European Medicines Agency for the disorder, BioMarin said.
The company plans to submit a supplemental application to U.S. regulators in the third quarter this year, followed by filings in Europe and other regions.
Voxzogo was already approved in 2021 in the U.S. for patients with achondroplasia, a form of dwarfism.
