FDA approves Hemlibra ® for haemophilia A patients

FDA approves Hemlibra

Roche announced today that the FDA approves Hemlibra ® (emicizumab-kxwh) for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adults and children, ages newborn and older, with haemophilia A without factor VIII inhibitors.

Hemlibra is now the only prophylactic treatment for people with haemophilia A with and without factor VIII inhibitors that can be administered subcutaneously (under the skin) and at multiple dosing options (once weekly, every two weeks or every four weeks). This approval is based on positive results from the phase III HAVEN 3 and HAVEN 4 studies.

Hemlibra prophylaxis led to statistically significant and clinically meaningful reductions in treated bleeds compared to no prophylaxis (primary endpoint) and across all other bleed-related endpoints in the HAVEN 3 study, and showed a clinically meaningful control of bleeding in the HAVEN 4 study.

“we now have a new class of medicine for the first time in nearly 20 years”

Haemophilia A is an inherited, serious disorder in which a person’s blood does not clot properly, leading to uncontrolled and often spontaneous bleeding. Haemophilia A affects around 320,000 people worldwide. Shire Pharmaceuticals recently released results from a UK patient survey which showed the needs of haemophilia patients.

Michael Callaghan, MD, haematologist, Children’s Hospital of Michigan, Detroit said, “Many preventative treatment options for people with haemophilia A without factor VIII inhibitors require intravenous infusions several times a week. Even then, people can still experience bleeds, and there has been a need for more treatment options. Now that the FDA approves Hemlibra this is an important advancement for the entire haemophilia A community, as we now have a new class of medicine for the first time in nearly 20 years. Hemlibra can reduce bleeds, and it offers a new subcutaneous administration once weekly, every two weeks or every four weeks.”

As the FDA approves Hemlibra, Sandra Horning, MD, Roche’s Chief Medical Officer and Head of Global Product Development comments, “The approval of Hemlibra reflects our commitment to groundbreaking science and the development of medicines with the potential to redefine the standard of care. Hemlibra is now the only FDA-approved medicine for people with haemophilia A with and without factor VIII inhibitors, based on the efficacy and safety profile demonstrated across four pivotal studies. We want to thank the haemophilia community for their partnership in helping us bring this new option to everyone living with haemophilia A.”

Earlier this year Roche reported positive results from the Hemlibra drug trial.