FDA Rejects Gene Therapy for Hemophilia A

Last Tuesday, the FDA rejected BioMarin Pharmaceutical’s gene therapy, Roctavian, for patients with hemophilia A. According to ABC News, the gene therapy infusion was meant to be a single treatment designed to prevent bleeding and eliminate the need for blood-clotting infusions.

Gene Therapy

Before we go into Roctavian, you may be wondering what gene therapy is. According to Genetics Home Reference, part of the U.S. National Library of Medicine, gene therapy is:

an experimental technique that uses genes to treat or prevent disease. In the future, this technique may allow doctors to treat a disorder by [replacing a mutated gene with a healthy copy, inactivating a mutated gene, or introducing a new gene into the body to help fight a disease.]

However, this type of treatment is generally only considered for diseases or disorders with no other cures. Many patients, doctors, and researchers were excited about BioMarin’s unique gene therapy. Every year, patients with severe hemophilia A require up to 150 FVIII infusions. Although this doesn’t always prevent painful bleeding, it reduces them. With Roctavian, an inactivated viral vector delivers a working gene to the body to stimulate the production of FVIII.

However, the FDA rejected the drug citing a concern that Roctavian might not be a singular and long-lasting treatment. This concern was supplemented by the potential price tag: up to $3m per dose. Before the gene therapy is approved, the FDA recommended completion of a late-stage patient study, alongside 2 years of follow-up data. As a result, potential approval for Roctavian may not occur until 2022 or 2023.

Hemophilia A

Also known as Factor VIII deficiency, hemophilia A is a rare genetic bleeding disorder. Patients with hemophilia A lack enough Factor VIII (FVIII), a blood clotting protein. This condition is rare within females. Around 20,000 U.S. citizens have hemophilia A.

Symptoms vary in severity. However, they may include:

  • Easy bruising and bleeding
  • Digestive and urinary tract bleeding
  • Excessive menstruation or bleeding following childbirth
  • Joint pain and swelling
  • Frequent nose bleeds
  • Excessive bleeding after trauma, dental work, surgery, or other injuries

Learn more about hemophilia A.

Jessica Lynn

Jessica Lynn

Jessica Lynn has an educational background in writing and marketing. She firmly believes in the power of writing in amplifying voices, and looks forward to doing so for the rare disease community.

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