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FDA Approves New Recombinant Factor Following LEOPOLD Trials

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Note: The following is an edited version of a press release from Bayer. The original press release can be found here


The U.S. Food and Drug Administration (FDA) has approved Bayer’s KOVALTRY® Antihemophilic Factor (Recombinant), an unmodified, full-length factor VIII compound for the treatment of hemophilia A in children and adults. The approval is based on results from the LEOPOLD (Long-Term Efficacy Open-Label Program in Severe Hemophilia A Disease) clinical trials, which supported the approval of KOVALTRY for routine prophylaxis to reduce the frequency of bleeding episodes. KOVALTRY can be used two or three times per week in adolescents and adults, and two or three times per week or every other day in children.

“In the LEOPOLD trials, KOVALTRY reduced bleeding episodes in patients with hemophilia A when infused twice to three times per week with routine prophylaxis,” said Sanjay P. Ahuja, MD, LEOPOLD investigator and Director, Hemostasis & Thrombosis Center at University Hospitals Rainbow Babies & Children’s Hospital and Associate Professor of Pediatrics at Case Western Reserve University School of Medicine in Cleveland, Ohio. “KOVALTRY may offer appropriate patients a twice-weekly prophylaxis dosing option.”

Hemophilia A, also known as factor VIII deficiency or classic hemophilia, is a largely inherited bleeding disorder in which one of the proteins needed to form blood clots in the body is missing or reduced. Hemophilia A is the most common type of hemophilia and is characterized by prolonged or spontaneous bleeding, especially into the joints, muscles or internal organs. It is estimated that 16,000 people are living with hemophilia A in the U.S. today. Treatment of hemophilia A involves infusing replacement factor into a vein.

Earlier this year, Bayer received approval of KOVALTRY in Europe and Canada. The company has submitted an application for approval of KOVALTRY in Japan, and plans to file for approval in other countries in the coming months.

“Hemophilia care has changed significantly in recent years, but we have a long way to go to help those living with hemophilia,” said Val Bias, CEO, the National Hemophilia Foundation. “It is vitally important that people are able to choose from a range of treatments, and we applaud Bayer for bringing forth new product options to help people manage their disease.

About LEOPOLD

The clinical trial program was designed to evaluate KOVALTRY pharmacokinetics, safety, efficacy of prophylaxis, treatment of bleeds and perioperative management in adults, adolescents, and children with severe hemophilia A.

LEOPOLD 1 was a multi-center, open-label, cross-over, uncontrolled study in adolescent and adult (age ≥12 years to

LEOPOLD 2 was a multi-center, open-label, cross-over, uncontrolled, randomized study in adolescent and adult (age ≥12 years to <65 years) PTPs evaluating the superiority of prophylaxis over on-demand treatment with KOVALTRY over a one-year treatment period. ABR was the primary efficacy variable.

LEOPOLD Kids Part A was a multi-center, open-label, uncontrolled study in pediatric (≤12 years of age) PTPs evaluating the pharmacokinetics, efficacy and safety of routine prophylaxis, and perioperative management of bleeding with KOVALTRY. The primary efficacy variable was annualized number of total bleeds during routine prophylaxis that occurred within 48 hours of previous prophylaxis infusion.

The most frequently reported adverse reactions in the clinical trials (>= 3%) were headache, pyrexia (fever), and pruritus (itchy rash).

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