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FDA Denies Biomarin's Gene Therapy. Shares Tank.



On August 18, 2020, The Food and Drug Administration (FDA) rejected a gene therapy application developed by Biomarin. The company announced that the regulatory agency issued a CRL (Complete Response Letter), showing that the application is not ready for approval. The gene therapy, known as Valoctocogene Roxaparvovec (Roctavian), was created for severe hemophilia A. Hemophilia A is an unusual blood disorder that is characterized by F8 genetic abnormalities that provides a process for making factor VIII. This protein plays a significant role in blood clot formation.


Doctors and patients widely accepted Roctavian. This is because the genetic modification was performed to fix the genetic defect that occurs in the bloodstream of patients with severe hemophilia A deficient or decreased clotting protein. FDA's endorsement would have been a significant accomplishment, marking decades of medical research that contributed to Roctavian and several other similarly functional new drugs. As such, the decision from the regulatory body came as a surprise to many stakeholders.


BioMarin is widely known as a company aimed at creating first-class therapies. With its innovation, the company's therapy provides treatment for patients living with severe life-threatening genetic disorders. Biomarin is well known for its speed and efficiency in research, development, sourcing, and market activities designed to deliver immediate treatments. In 2015, BioMarin was ranked 10th on Forbes' list of innovative companies. A primary news source for global clinical studies, known as CenterWatch, listed Biomarin as one of the industry's top product producers.


BioMarin requested data from two medical studies to back up its Roctavian endorsement demand. Findings from a lesser, initial study showed medication in the 13 patients receiving large doses of Roctavian almost removed bleeds, liberating them from common drugs to treat their disease. Four years later, the results of Roctavian continue to hold up. A critical evaluation of a broader Phase 3 trial was intended to support these early outcomes since BioMarin had not tracked the research participants for so long.


But in a report to BioMarin, the regulatory body said that the research would require two-year follow-up data from a Roctavian Phase 3 study. California biotech estimates that the last participant in that testing will fulfill two years of study in late 2021. This means that the company will likely set up a re-approval demand and a mid-or late-2022 FDA review. In conclusion, BioMarin noted that it would consult with the FDA to explore the next measures in the following weeks. Due to the news reported on Wednesday, the company lost a third of its shares the next day.

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