NEW YORK (TheStreet) -- BioMarin Pharmaceuticals (BMRN) shares are up 11.73% to $138.11 in early market trading on Thursday after posting positive Phase II data from its achondroplasia (dwarfism) treatment.
The study was relatively small, involving only 26 children, but there are no current treatments for the disorder currently and the trials showed a mean increase of 50% in their annual growth.
The treatment has already been granted "orphan drug" designation from the Food and Drug Administration, given to treatments for rare conditions.
"We are very encouraged to have observed evidence of activity with vosoritide in children participating in our Phase 2 study. In children receiving the highest dose of 15 micrograms per kilogram daily, we observed a 50% increase in mean annualized growth velocity compared to their own natural history control growth velocity. This increase in growth velocity, if maintained, could allow children with achondroplasia to resume a normalized growth rate," said company Vice President Wolfgang Dummer, M.D., Ph.D.
TheStreet Ratings team rates BIOMARIN PHARMACEUTICAL INC as a Hold with a ratings score of C. TheStreet Ratings Team has this to say about their recommendation:
Highlights from the analysis by TheStreet Ratings Team goes as follows:
- You can view the full analysis from the report here: BMRN Ratings Report