Benzinga - Biomarin Pharmaceutical Inc (NASDAQ: BMRN) shares are trading higher by 6.97% to $97.54 Wednesday morning after the company announced advancements in the FDA review of ROCTAVIAN for adults with severe hemophilia A.
What Else?
In addition to the RMAT Designation and Breakthrough Therapy Designation, BioMarin says valoctocogene roxaparvovec also received orphan drug designation from the EMA and FDA for the treatment of severe hemophilia A.
Orphan drug designation is reserved for medicines treating rare, life-threatening, or chronically debilitating diseases. The European Commission (EC) granted conditional marketing authorization to valoctocogene roxaparvovec gene therapy under the brand name ROCTAVIAN™ on August 24, 2022.
"The review of a BLA is a dynamic process, and we appreciate FDA's ongoing engagement as we work toward delivering a potentially transformative treatment choice to those patients with severe hemophilia A," said Hank Fuchs, M.D., President of Worldwide Research and Development at BioMarin. "We look forward to further dialogue with the Agency as it reviews our application."
According to data from Benzinga Pro, BMRN has a 52-week high of $98.62 and a 52-week low of $70.73.
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