FDA expands indication of Roche's Hemlibra in haemophilia

The FDA has chosen to expand the label of Roche’s Hemlibra (emicizumab-kxwh), approving it for routine prophylaxis to prevent the frequency of bleeding episodes in all adults and children with haemophilia A without subcutaneous factor VIII inhibitors, it has emerged.

The expansion covers several dosing options - once weekly, every two weeks or every four weeks – and is based on the findings of two Phase 3 trials which demonstrated that Hemlibra led to "statistically significant and clinically meaningful reductions in treated bleeds compared to no prophylaxis", meeting its primary endpoint. The drug also showed "a clinically meaningful control of bleeding".

Specifically, those taking the treatment once weekly or fortnightly saw a 96-97% reduction in treated bleeds compared to those who received no prophylaxis. According to Roche, the therapy is the first to significantly reduce treated bleeds compared to standard of care, prior factor VIII prophylaxis.

"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," explained Dr Michael Callaghan, Haematologist at Children’s Hospital of Michigan in Detroit. "The approval of Hemlibra 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."

Dr Sandra Horning, Roche’s Chief Medical Officer and Head of Global Product Development, commented: "Today’s 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."

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