Chugai Pharmaceutical has announced that the US Food and Drug Administration (FDA) has approved the bispecific antibody emicizumab (US product name: Hemlibra) for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and paediatric patients with haemophilia A (congenital factor VIII deficiency) with factor VIII inhibitors. This represents the first regulatory approval for Hemlibra around the world. The US Biologics License Application (BLA) was submitted by Genentech, a member of Roche Group.

“We are pleased that Hemlibra, a biopharmaceutical created by Chugai scientists based on their exceptional idea, has received its first regulatory approval,” said Chugai’s president & COO, Tatsuro Kosaka. “Hemlibra is a first in class biopharmaceutical which is an advancement on the current limited treatment options available for patients with haemophilia A with inhibitors. We anticipate that patients will receive significant potential benefits from Hemlibra, which enables once-weekly subcutaneous injection as well as reduction of the bleeding risk.”

This regulatory approval is based on the data from two clinical studies, the results of HAVEN 1 study (NCT02622321) and the interim analysis of HAVEN 2 study (NCT02795767) in patients with haemophilia A with factor VIII inhibitors. HAVEN 1 included adult and adolescent patients and HAVEN 2 included paediatric patients, and were both conducted under a collaboration between Chugai, Roche and Genentech. In the HAVEN 1 study, adults and adolescents with haemophilia A with inhibitors who received Hemlibra prophylaxis (n=35) had a statistically significant and clinically meaningful 87% (95% CI: 72.3-94.3, p<0.0001) reduction in treated bleeds compared with those who received no prophylaxis (n=18). Interim results from the HAVEN 2 study, paediatric patients with haemophilia A with inhibitors showed that 87% of patients (95% CI: 66.4-97.2) who received Hemlibra prophylaxis (n=23) experienced zero treated bleeds. The most common adverse events (AEs) occurring in 10% or more of people treated with Hemlibra in pooled studies were injection site reactions, headache and joint pain (arthralgia).

Hemlibra was reviewed by the FDA under Priority Review. It was granted Breakthrough Therapy Designation by the FDA in adults and adolescents with haemophilia A with inhibitors in September 2015. Outside the US, emicizumab has been filed in the EU in June 2017 and data from HAVEN 1 and HAVEN 2 are being reviewed under Accelerated Assessment by the European Medicines Agency. In Japan, the drug received orphan drug designation by the Ministry of Health, Labour and Welfare for the prevention and reduction of bleeding episodes in patients with congenital FVIIl deficiency (haemophilia A) who developed inhibitors to FVIII in August 2016, and a new drug application was filed in July 2017.