U.S. health regulators have approved a gene therapy for the most common form of hemophilia.
This photo provided by BioMarin in June 2023 shows a vial and packaging for their drug Roctavian. U.S. officials on Thursday, June 19, 2023 approved the drugmaker's gene therapy for the most common form of hemophilia, an infused treatment that can significantly reduce dangerous bleeding problems. U.S. officials on Thursday approved drugmaker BioMarin's gene therapy for the most common form of hemophilia, a $2.
BioMarin said in a statement that the FDA approval was based on a three-year study showing a 50% reduction in annual bleeding incidents among 134 patients who received the treatment. Most patients continued to respond to the treatment beyond three years, without needing regular IV infusions, the company said.
BioMarin said it estimates about 2,500 U.S. patients will be eligible to receive the therapy under the terms of the FDA's approval. Patients with certain underlying health problems and conditions are excluded under the prescribing information. Dr. Margaret Ragni called Roctavian “a major improvement in terms of reducing the burden of disease.” But she notes that many patients are comfortable with their current treatments and may be hesitant to try a new gene therapy.
The San Rafael, California-based company excluded patients with certain potentially complicating conditions, including liver disorders and resistance to the standard blood clotting protein, which often develops in some hemophilia patients. BioMarin’s president for research and development, Dr. Henry Fuchs, said the company is conducting studies in some of those excluded groups to see if they can safely receive the therapy.
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