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Breakthrough haemophilia treatment wins prestigious drug discovery award

The first novel prophylactic medicine in nearly 20 years to treat people with haemophilia with or without Factor VIII inhibitors has been named Drug Discovery of the Year 2019. The team of researchers from Roche, Chugai and Genentech responsible for the development of Hemlibra® (emicizumab in the EU and emicizumab-kxwh in the US) have been recognised with the British Pharmacological Society’s prestigious annual award for the drug’s effectiveness in substantially reducing bleeds – in the majority of cases to zero – in both children and adults.

Hemlibra® is a bispecific factor IXa- and factor X-directed antibody. It works by mimicking the missing protein coagulation function of factor VIII, which causes haemophilia A. Factor VIII is needed by the body to stop bleeding and to start the healing process in wounds, dramatically improving the quality of life for the patient.

Haemophilia is a rare genetic deficiency that affects the blood's ability to form a clot. In the majority of cases it is inherited and mainly affects males. Haemophilia A is the most common type of haemophilia, affecting about one in every 10,000 males. There is currently no cure. Hemlibra® offers a breakthrough therapy for patients and the research team behind it have made a significant contribution to treatment options.

The Roche-Chugai-Genentech team were commended by an international judging panel for the successful translation of Hemlibra® from preclinical development to clinical studies and approval as a first-in-class medicine. Their success is a pioneering example of the role of pharmacology in the drug discovery and development process.

Hemlibra’s® use has already had a significant impact in redefining the current standard of care for haemophilia patients. Many preventive treatment options for people with haemophilia A with or without Factor VIII inhibitors require intravenous infusions several times a week. This new class of medicine offers a new subcutaneous route of administration once weekly (EU), or weekly, every two weeks or every month (US).

Dr Tom Blackburn, Chair of the Society’s Industry Sub-Committee, said:

“Hemlibra® represents a major breakthrough therapy for this life-threatening disorder and redefines the current standard of care.”

Dr Alex Phipps, Head of Clinical Pharmacology – Oncology at Roche, said:

“Given the quality of the medicines that have received this prestigious award, we are both delighted and humbled that Hemlibra® should be the 2018 recipient.
“Hemlibra® was developed following an exhaustive search to discover the antibody best structurally suited to mimicking Factor VIII. The early results in a small cohort of Japanese haemophilia patients indicated that it might offer a completely new, efficacious and convenient treatment option and Roche/Genentech developed the medicine with this in mind.
“We are hopeful that the focus on pharmacological excellence that led to Hemlibra® will now offer patients an opportunity to lead productive lives with their condition held firmly in check.”

The Drug Discovery of the Year Award will be presented to the Roche team by Professor Steve Hill, President of the British Pharmacological Society, at a prize-giving ceremony on 19 December as part of the Society’s annual conference, Pharmacology 2018.


About Drug Discovery of the Year

The British Pharmacological Society established the Drug Discovery of the Year in 2012 to highlight the crucial role pharmacology plays in the development of new medicines. The Society’s Industry Sub-Committee selected Hemlibra® from a shortlist of four excellent first-in-class drugs and outstanding presentations.

Published: 19 Dec 2018 in Society news