FDA approves the first new drug in decades to reduce bleeding in hemophilia patients; annual costs could reach $700,000 per patient


The U.S. Food and Drug Administration (FDA) recently approved the first new treatment in nearly two decades to prevent internal bleeding in some patients with hemophilia A.

Hemlibra (emicizumab-kxwh) prevents or reduces the frequency of bleeding episodes in adult and pediatric patients with hemophilia A who have developed antibodies called Factor VIII inhibitors.

About 20,000 Americans — mostly males — have hemophilia, an inherited, potentially life-threatening blood-clotting disorder, according to the National Institutes of Health. An estimated 80 percent of hemophilia patients have hemophilia A. Patients with hemophilia A can experience repeated episodes of serious bleeding. In severe cases, repeated bleeding in the joints can cause intense pain and lead to problems walking.

While the hemophilia patient population is relatively small, the cost of hemophilia products for patients with factor VIII inhibitors is high. The annual cost of Hemlibra may range from nearly $200,000 to $700,000 based on patient weight.

Drug Overview and Place in Therapy

Hemlibra offers novel treatment for hemophilia A patients who have developed resistance to standard medicines for preventing and reducing bleeding episodes.

Patients with hemophilia A are missing a gene that produces Factor VIII, a protein that enables blood to clot. Nearly one-third of patients with severe hemophilia A develop an immune response known as a FVIII inhibitor or antibody, which interferes with the effectiveness of currently available treatments for hemophilia.

Hemlibra is a first-in-class therapy that bridges two other factors in the blood to restore blood clotting for these patients, negating the need for factor VIII. In two clinical trials that led to approval of the drug, Hemlibra reduced the number of bleeding episodes that required treatment.

Hemlibra will compete with Feiba (anti-inhibitor coagulant complex) and NovoSeven (coagulation factor VIIa recombinant), both indicated for patients with hemophilia A with factor VIII inhibitors. NovoSeven, however, does not have an indication for routine bleeding prophylaxis.

Given its subcutaneous once weekly dosing and option for self-injection, Hemlibra offers potential to improve patient care and convenience, in comparison to current treatment options that often require frequent intravenous infusions.

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Forward-Thinking Strategies to Manage Drug Costs

MedImpact’s Emerging Therapeutics Strategy team provides up-to-date pipeline surveillance that can help your health plan prepare for Hemlibra and other high-impact drugs. The Emerging Therapeutics Strategy group also works closely with MedImpact’s Clinical Formulary team to identify advanced solutions that can help lower drug costs and improve member care.

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