Roche’s Hemlibra approved in expanded haemophilia A use

Roche’s Hemlibra has been approved in an expanded use in haemophilia A in the US, as the Swiss firm seeks to build a new blockbuster franchise in rare diseases.

The firm is best known for its ‘big three’ cancer blockbusters – Avastin, Herceptin, and Rituxan – but these are all threatened by cheaper biosimilar competition as their patents have run out or are close to expiring.

While Roche is still involved in cancer drugs, it is diversifying into other disease areas and has targeted the haemophilia market with Hemlibra (emicizumab).

With this new indication, Hemlibra is approved 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.

It is already approved in haemophilia in patients inhibitors – antibodies that block the function of factor VIII replacement therapies.

Roche is competing against established firms such as Shire, which produce factor VIII replacement therapies requiring regular injections.

In order to win over patients and doctors who are risk-averse and loyal to established treatments, Roche has formulated the drug so patients can administer it themselves subcutaneously every week, every fortnight, or every four weeks.

Michael Callaghan, haematologist, at the 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.

“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.”

Roche’s Sandra Horning

Sandra Horning, Roche’s chief medical officer, said: “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”.


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