Esperoct Available in US to Treat Adults and Children with Hemophilia A, Novo Nordisk Says

Esperoct (turoctocog alfa pegol) is now available in the U.S. to treat and control bleeding in adults and children with hemophilia A, the therapy’s manufacturer, Novo Nordisk, announced.

The treatment’s use was approved by the U.S. Food and Drug Administration (FDA) in February 2019, but its availability required third-party intellectual property agreements that have now been secured.

Hemophilia A is caused by the lack of a functional blood clotting factor, called factor VIII (FVIII). Esperoct, formerly known as N8-GP, is a recombinant (lab-made) version of FVIII that has been engineered to have an extended half-life in order to remain active in the body for longer periods of time than the unmodified protein. The half-life of a medicine refers to the time it takes for the levels of a compound to drop by half.

As such, Esperoct may maintain stable FVIII levels for longer periods of time than other FVIII replacement therapies, requiring less frequent dosing to prevent spontaneous bleeding episodes.

“At Novo Nordisk, we have a longstanding commitment to the hemophilia community, beginning with the development of our first recombinant factor therapy more than 30 years ago,” Pia D’Urbano, corporate vice president of biopharmaceuticals at Novo Nordisk, said in a press release.

“We recognize that people with rare bleeding disorders need multiple therapy options and are proud to now make Esperoct available as a new treatment option for those living with hemophilia A, the most common form of the chronic bleeding disorder,” D’Urbano added.

Esperoct is approved in the U.S. for both prophylactic (preventative) and on-demand control of bleeding episodes in adults and children with hemophilia A.  Its use by patients for these same reasons is also approved in Canada and the European Union.

The FDA based Esperoct’s approval on data from a series of Phase 3 clinical trials (NCT01731600, NCT01480180, NCT01489111, NCT03528551, and NCT02137850), which included 270 children and adults with severe hemophilia A.

Their results showed that Esperoct maintained low annualized bleeding rates (ABRs) in children under 12 (median ABR, 2.0) and in adults and adolescents age 12 and older (median ABR, 1.2) when given in fixed dosing schedules. Approved use for routine prophylaxis is 65 IU/kg twice weekly for children up to age 12 and 50 IU/kg every four days for adults and teenagers, with adjustments to be made based on bleeding episodes.

Treatment was also found to be safe and well-tolerated. The most common side effects include rash, redness, itching and reactions at the injection site.

Additional studies have shown that Esperoct can stabilize or improve patients’ health-related quality of life.

Novo Nordisk offers a product assistance program for people who have been prescribed Esperoct or other company treatments. This includes providing the medication free-of-charge to eligible patients in the U.S. who lack insurance and have a qualifying low family income.