By Christiana Sciaudone
Investing.com -- BioMarin Pharmaceutical Inc (NASDAQ:BMRN) slumped more than 36% on Wednesday after the U.S. Food and Drug Administration rejected its gene therapy for hemophilia A.
It's not the end of the world for the drug, though. The FDA cited the need for longer-term data, which could push approval -- if it comes at all -- to 2022.
All signs pointed to approval this month after clinic trials showed it reduced the bleeding rate of patients with the disorder, decreasing or eliminating the need for treatment, Reuters reported.
The market for the drug is not especially large. There are about 16,000 patients in the U.S. with hemophilia A in which they are missing the factor VIII clotting protein. The gene therapy would allow them to forego standard therapy requiring patients to infuse factor proteins into their bloodstreams several times a week for life, costing hundreds of thousands of dollars a year, according to Reuters.
BioMarin had said the gene therapy could be priced in a range of $1 million to $3 million, making it the most expensive one-time treatment, Reuters said.
The stock has 17 buy ratings, four holds and no sells.