BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) stock was on the rise today following positive news for its gene therapy efforts.
BioMarin Pharmaceutical Inc. announced its most recent results for valoctocogene roxaparvovec at the 2017 American Society of Hemophilia event over the weekend. Valoctocogene roxaparvovec is a treatment for severe hemophilia.
“We are very encouraged that our findings to date suggest a one-time infusion of valoctocogene roxaparvovec has the potential to eliminate bleeds, the need for exogenous factor VIII infusions and achieve FVIII levels in the normal range for patients with severe hemophilia A, with a very acceptable safety profile,” Hank Fuchs, President of Worldwide Research and Development at BioMarin Pharmaceutical Inc., said in a statement.
BioMarin Pharmaceutical Inc. notes that its treatment for severe hemophilia performed well in all patients, even those that were subject to the lowest dosage. It points out that negative side effects weren’t enough to cause any of its patients to drop out of the study.
The most common side effects in the valoctocogene roxaparvovec study were alanine aminotransferase elevation, arthralgia, aspartate aminotransferase elevation, headache, back pain, fatigue and upper respiratory tract infection. Two patients in the study also suffered severe adverse effects, but only one was connected to the treatment and that person recovered.
BioMarin Pharmaceutical Inc.’s valoctocogene roxaparvovec treatment has been given Breakthrough Therapy Designation by the U.S. Food and Drug Administration. The treatment is also part of the European Union’s Priority Medicines imitative.
BMRN stock was up 6% as of noon Monday, but is down 7% year-to-date.