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BioMarin Announces Advancements in FDA Review of ROCTAVIAN™ (Valoctocogene Roxaparvovec) for Adults with Severe Hemophilia A
FDA No Longer Plans to Hold an Advisory Committee Meeting, as Previously Planned, to Discuss the Biologics License Application (BLA) BioMarin Remains on Track
About this update from Biomarin Pharmaceutical Inc.
[{"type":"text","content":"FDA No Longer Plans to Hold an Advisory Committee Meeting, as Previously Planned, to Discuss the Biologics License Application (BLA)\nBioMarin Remains on Track to Host Scheduled Manufacturing Inspections by FDA in the Coming Weeks\nSAN RAFAEL, Calif., Nov. 23, 2022 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced advancements in the U.S. Food and Drug Administration (FDA) review of the Biologics License Application (BLA) of ROCTAVIAN™ (valoctocogene roxaparvovec AAV gene therapy) for adults with severe hemophilia A. The Company was recently notified by the FDA that after further consideration, at this time, the Agency no longer plans to hold an advisory committee meeting to discuss the BLA for ROCTAVIAN that is currently under review. Previously, the FDA communicated to the Company that it did intend to hold an advisory committee meeting but did not specify a date. The Company also remains on track to host the scheduled FDA Pre-Licensure Inspection (PLI) of BioMarin's gene therapy manufacturing facility located in Novato, CA. \n\n \n \n \n \n \n \n\n \n\"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.\"\nAbout valoctocogene roxaparvovec (ROCTAVIAN™)\nThe FDA granted Regenerative Medicine Advanced Therapy (RMAT) designation to valoctocogene roxaparvovec in March 2021. RMAT is an expedited program intended to facilitate development and review of regenerative medicine therapies, such as valoctocogene roxaparvovec, that are expected to address an unmet medical need in patients with serious conditions. The RMAT designation is complementary to Breakthrough Therapy Designation, which the Company received for valoctocogene roxaparvovec in 2017.\nIn addition to the RMAT Designation and Breakthrough Therapy Designation, BioMarin's 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 Commissio...