Shares of the biotech drugmaker surged early Mon. after reporting positive late-stage trial data for its GALNS drug candidate to treat people with Morquio syndrome, a rare genetic disorder that causes problems like abnormal heart and skeletal development. BioMarin Pharmaceutical (BMRN) said patients displayed improved breathing, walking and stair-climbing after 36-48 weeks of treatment. ThinkEquity estimates a market opportunity of $575 mil a year. Shares soared 31.2% to 49.07.
- Pharmaceuticals & Drug Trials
- BioMarin Pharmaceutical
- Morquio syndrome