Chugai Pharmaceutical Co., Ltd.,one of Japan's leading research-based pharmaceutical companies, announced that Roche has received notification that the EU Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion for Hemlibra (emicizumab) for routine prophylaxis of bleeding episodes in people with haemophilia A with factor VIII inhibitors. The CHMP has recommended Hemlibra for use in all age groups.

"Following the regulatory approval from the US Food and Drug Administration (FDA) in November 2017, we are thrilled that based on the CHMP opinion Hemlibra is expected to be approved by the European Commission in the near future," said Chugai's senior vice president, head of project & lifecycle management unit, Dr. Yasushi Ito. "Existing treatments for people with hemophilia A with inhibitors need frequent intravenous injection every week, which remains a burden for both patients and their caregivers. Hemlibra has the benefit of enabling once-weekly subcutaneous (under the skin) injection. We are committed to deliver the drug to patients as early as possible in collaboration with Roche."

This positive opinion is based on the two pivotal studies for haemophilia A with inhibitors: the results of HAVEN1 study (NCT02622321) for adolescents and adults, and the interim analysis of HAVEN2 study (NCT02795767) in children.

Emicizumab is a bispecific monoclonal antibody, which was developed using Chugai's proprietary antibody engineering technologies. The drug is designed to bind factor IXa and factor X. In doing so, emicizumab provides the cofactor function of factor VIII in people with haemophilia A, who either lack or have impaired coagulation function of factor VIII1, 2). In November 2017, the drug (US product name: Hemlibra; Genentech) was approved by the FDA 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." An EU marketing authorization application was submitted in June 2017 and was reviewed under Accelerated Assessment by the European Medicines Agency. In Japan, emicizumab obtained an orphan drug designation in August 2016 from the Ministry of Health, Labour and Welfare for the prevention and reduction of bleeding episodes in patients with congenital factor VIII deficiency (haemophilia A) who developed inhibitors to factor VIII, followed by an application for regulatory approval filed in July 2017.

Chugai Pharmaceutical is one of Japan's leading research-based pharmaceutical companies with strengths in biotechnology products. Chugai, based in Tokyo, specializes in prescription pharmaceuticals and is listed on the 1st section of the Tokyo Stock Exchange.