- Biomarin Pharmaceutical Inc BMRN shares are falling in reaction to positive data from BridgeBio Pharma Inc’s BBIO dwarfism drug, heating the competition.
- At the highest dose level evaluated to date (Cohort 5, 0.25 mg/kg once daily), the mean increase from baseline in annualized height velocity (AHV) for the ten children with six-month visits was +3.03 cm/yr.
- 80% of the ten children with six-month visits were responders, with a change from baseline AHV of at least 25%. Among the responders, the average change from baseline in AHV was +3.81 cm/yr.
- BioMarin’s Voxzogo (vosoritide) became the first drug to win FDA approval in achondroplasia or short-limbed dwarfism space.
- The therapy was OK’ed in November 2021. In BioMarin’s pivotal study, patients on the drug grew an average of 1.57 centimeters taller per year.
- In 2022, BioMarin generated $169.1 million in Voxzogo sales; for FY23, the company forecasts $330 million – $380 million.
- Price Action: BMRN shares are down 7.27% at $93.84 on the last check Monday.