Washington, United States: The U.S. Food and Drug Administration approves Roctavian by BioMarin as a single does gene therapy for patients suffering with hemophilia A, which is a life-threatening hereditary bleeding disorder. For a single infusion, Roctavian will cost USD2.9 million. According to BioMarin, under the terms of approval by the FDA, approximately 2,500 patients in the United States are expected to be eligible for treatment, excluding patients with certain underlying health problems. The Roctavian uses an inactive virus, developed in the laboratory, for transposal of a substitute gene to liver cells that produce clotting proteins. Patients may also be able to produce their own protein once the treatment has been successful. There is a warning on the label that there may be uncommon and serious allergic reactions.

Further, the approval was based on a global study assessing safety and effectiveness of the medicine in adult men aged 18 to 70 years who had received FVIII replacement therapy before. For a minimum period of 3 years, 112 patients were followed. There was a much smaller number of bleeding events in those receiving treatment from the study. The mean annual rate of bleeding has fallen from 5.4 to 2.6 times per year. Corticosteroids have also been used to control the immune system in most patients treated with Roctavian. The Food and Drug Administration stated that over time, the response to treatment can decrease.

According to the Director of the FDA's Center for Biologics Evaluation and Research, “Hereditary hemophilia A is a potentially serious bleeding disorder. Severe cases of hemophilia A can cause life-threatening health issues due to increased risk of uncontrolled bleeding. Today's approval represents an important advance in providing treatment options for patients with this bleeding disorder, and treatment with gene therapy may reduce the need for ongoing routine therapy”. Further,  the Chairman and Chief Executive Officer of BioMarin stated that, “Today's approval of ROCTAVIAN builds on BioMarin's proven track record of advancing treatments that target the underlying cause of life-threatening genetic conditions, which has produced eight best- or first-in-class commercial therapies. We are proud to now offer adults with severe hemophilia A, a one-time, single-dose treatment option. We are especially grateful to the bleeding disorders community for its support of this program, and to all the patients and healthcare providers who participated in our clinical trials."

According to TechSci Research, Mutations that make it impossible to manufacture proteins needed for blood clotting are responsible for hemophilia. Hemophilia A is the most severe variant of the condition, and some patients can experience spontaneous bleeding even without any injury. If left untreated, the condition may lead to bleeding in joints and organs, including the brain. Increasing approvals by FDA for the new launches is boosting the market for gene therapy, which is a remarkable step for improving the medicine healthcare system.