FDA Approves ESPEROCT For Adult, Pediatric Hemophilia A

The US Food and Drug Administration (FDA) has approved the Biologics License Application for turoctocog alfa pegol (ESPEROCT) for the treatment of adults and children with hemophilia A.

ESPEROCT, which was assessed under the investigative name N8-GP, is now approved of routine prophylaxis to reduce the frequency of bleeding episodes, as well as on-demand treatment and control of bleeding episodes in patients with congenital factor VIII (FVIII) deficency.

The Novo Nordisk-based drug has been granted approved with supporting evidence from a 270-patient assessment—the largest pre-registration clinical program ever conducted in the hemophilia A field, according to the company. In the trial, previously treated people (PTPs) with severe hemophilia A and at least 5 years of clinical exposure were treated with a fixed-dose regimen of 1 injection every 4 days (adults and adolescents) or 3-4 days twice-weekly (children).

The clinical program spanned 5 prospective, multi-center clinical trials and involved 202 (74.8%) adults and 68 (25.2%) children. All patients had no history of inhibitor use, and were assessed for a total of 889 patient years of ESPEROCT therapy.

Investigators reported that patients treated with 50 IU/kg every 4 days had effective prophylaxis, and a median annual bleed rate (ABR) of 1.18. The therapy was also found to be efficacious in both treating and controlling bleeding episodes, as well as in situations of perioperative management. It was well tolerated across all patient age groups, and no safety concerns nor were reported. ESPEROCT’s safety profile was reported as similar to that of other long-acting products designated for FVIII.

Though Novo Nordisk will be unable to launch the therapy in the US before 2020 due to third-party intellectual property agreements, Mads Krogsgaard Thomsen, the company’s executive vice president and chief science officer, expressed excitement for ESPEROCT’s approval, and described it as among an “important expansion of the treatment options” made available by Novo Nordisk for hemophilia A.

"We are confident that ESPEROCT will provide people with hemophilia A a less burdensome and simple, fixed dosing regimen for prophylaxis and treatment of bleeding episodes, resulting in improved quality of life,” Thomsen said in a statement.