The U.S. Food and Drug Administration (FDA) has approved Helimbra (Emicizumab-kxwh) for preventing or reducing bleeding episodes in some patients of Hemophilia A.
People with Hemophilia A generally lack a gene that is responsible for producing blood-clotting factor, called Factor VIII. This drug is administered in patients with antibodies, known as Factor VIII inhibitors.
According to FDA, approximately 5000 babies born in the U.S. are affected by hemophilia, and out of these, 80% have Hemophilia A. Also, this disease is more common in male babies.
In Hemophilia A, patients generally experience bleeding in the joints, which can cause severe damage. In some cases, the patients start developing antibodies against the standard treatment for bleeding episodes.
Dr. Richard Pazdur, Acting Director, FDA’s Office of Hematology and Oncology Products, told that the approval for Helimbra is a great preventive measure to prevent bleeding episodes in patients with hemophilia A and Factor VIII inhibitors. He also added that when patients were treated with Helimbra, they showed improvement in their physical activities.
A few side effects of the drug was also reported like reaction on the site of injection, headache and joint pain.
FDA said, “The drug’s label includes a boxed warning of possible blood clots among recipients who also are given anti-bleeding medication.”