Hemophilia A is an inherited blood-clotting disorder that can lead to excessive bleeding, even from minor injuries. According to a recent ABC News article, the FDA has granted approval to BioMarin for its gene therapy called Roctavian, designed to treat severe cases of hemophilia A. Roctavian is an infused treatment that aims to reduce dangerous bleeding problems associated with the condition. It’s the first gene therapy approved for patients with severe hemophilia A.
The FDA's approval was based on a three-year study involving 134 patients, which showed a 50% reduction in annual bleeding incidents. BioMarin claims most patients continued to respond positively to the treatment after three years, eliminating the need for regular intravenous infusions. However, the therapy may have rare, severe allergic reactions as potential side effects. BioMarin estimates that around 2,500 eligible patients in the United States could benefit from the treatment, which has a price tag of $2.9 million.