FDA Approves Alhemo® Injection for Daily Bleeding Prevention in Hemophilia A or B with Inhibitors

Novo Nordisk has announced a significant advancement for individuals living with hemophilia A or B with inhibitors. The U.S. Food and Drug Administration (FDA) has approved Alhemo® (concizumab-mtci), a subcutaneous injection administered once daily, to prevent or reduce bleeding episodes in adults and children aged 12 and older. This approval highlights Novo Nordisk's dedication to addressing rare bleeding disorders, extending a commitment that spans over 35 years.

Alhemo® operates as a tissue factor pathway inhibitor (TFPI) antagonist, delivered through a prefilled, premixed pen designed for subcutaneous injection. This method presents a less invasive alternative compared to the traditional intravenous infusions commonly used in current treatments for hemophilia A or B with inhibitors. The introduction of Alhemo® as the first subcutaneous treatment of its kind is a pioneering development for this patient demographic.

The drug works by inhibiting TFPI, a protein that prevents blood clotting, thereby improving thrombin production. Thrombin is crucial for blood clot formation, especially when other clotting factors are deficient or absent due to inhibitors. The efficacy of Alhemo® was demonstrated in the pivotal Phase 3 clinical trial, explorer7, which confirmed an 86% decrease in treated spontaneous and traumatic bleeds among patients using Alhemo® prophylaxis compared to those without prophylaxis.

The explorer7 trial involved adult and pediatric patients over 12 years, establishing the safety and efficacy of Alhemo®. Participants receiving Alhemo® showed a significantly lower annual bleeding rate (ABR) compared to those not on prophylaxis. Specifically, the estimated mean ABR for patients on Alhemo® was 1.7, compared to 11.8 for those without prophylaxis. Additionally, 64% of those on Alhemo® experienced no treated spontaneous or traumatic bleeds in the first 24 weeks of treatment, a stark contrast to the 11% in the no-prophylaxis group.

The FDA's approval of Alhemo® marks a breakthrough in hemophilia treatment, particularly for those with inhibitors, who face a tougher challenge in managing their condition. Inhibitors develop in approximately 30% of individuals with severe hemophilia A and 5-10% with severe hemophilia B, complicating the treatment process. For hemophilia B patients with inhibitors, treatment options have been limited, underscoring the significance of this new therapy.

Anna Windle, Senior Vice President of Clinical Development at Novo Nordisk, emphasized the approval as a transformative moment for patients with limited options, reaffirming the company's commitment to innovative treatments for rare diseases. The introduction of Alhemo® offers a much-needed alternative to the standard care options, providing more personalized treatment opportunities.

Beyond the U.S., Alhemo® has received approval in several countries, including Australia, Japan, Switzerland, and throughout the European Union, with specific indications varying by region.

Hemophilia is a rare condition affecting the blood's ability to clot, impacting around 32,000 people in the U.S. alone. It is caused by genetic mutations affecting clotting factor proteins, leading to bleeding issues. Traditional treatments involve replacing these proteins through intravenous infusions, but inhibitors can render these therapies ineffective, limiting treatment options.

Alhemo® provides a new avenue for managing hemophilia with inhibitors by enhancing factor Xa production and thrombin generation, crucial for effective blood clotting. Its approval marks a significant development in offering patients a reliable and less invasive treatment method, potentially transforming care standards for those living with hemophilia A or B with inhibitors.