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FDA Approves Breakthrough Gene Therapy for Severe Hemophilia A in Adult Patients

Saturday, July 01, 2023

The US Food and Drug Administration (FDA) has officially approved Roctavian, a groundbreaking gene therapy utilizing adeno-associated virus vectors, for the treatment of severe hemophilia A in adult patients who do not have pre-existing antibodies to adeno-associated virus serotype 5, as confirmed by an FDA-approved test.

Hemophilia A is an inherited bleeding disorder caused by a mutation in the gene responsible for producing factor VIII (FVIII), a vital protein for blood clotting. Severe cases of hemophilia A can lead to life-threatening complications due to an increased risk of uncontrolled bleeding. This newly approved gene therapy, Roctavian, represents a significant milestone in offering treatment options for individuals with this bleeding disorder. Moreover, gene therapy treatments have the potential to reduce the need for ongoing routine therapies, according to Dr. Peter Marks, director of the FDA's Center for Evaluation and Research of Biological Products.

Roctavian is administered as a single dose via intravenous infusion and utilizes a viral vector to deliver a gene responsible for producing coagulation factor VIII. By expressing this gene in the liver, Roctavian aims to elevate factor VIII levels in the blood, thereby reducing the risk of uncontrolled bleeding.

To evaluate the safety and effectiveness of Roctavian, a multinational study was conducted involving adult men between the ages of 18 and 70 who had severe hemophilia A and had previously received factor VIII replacement therapy. Efficacy was established based on data from a cohort of 112 patients who were followed for a minimum of three years after receiving Roctavian. Results showed a decrease in the average annualized bleeding rate from 5.4 bleeds per year at baseline to 2.6 bleeds per year. It's important to note that most patients who received Roctavian also received corticosteroids to suppress the immune system, ensuring the therapy's efficacy and safety. However, the response to Roctavian treatment may diminish over time.

Common adverse reactions associated with Roctavian include mild changes in liver function, headache, nausea, vomiting, fatigue, abdominal pain, and infusion-related reactions. Close monitoring for infusion-related reactions and liver enzyme elevation is recommended during Roctavian administration. In some cases, Roctavian treatment led to increased factor VIII activity levels beyond normal limits, which can increase the risk of thromboembolic events (blood clots that can obstruct blood flow and cause damage). While there is a theoretical risk of developing hepatocellular carcinoma (liver cancer) or other types of cancer associated with the introduction of the DNA sequence from Roctavian, no such cases were observed in clinical studies.

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