FDA grants breakthrough status to Roche's Hemlibra in haemophilia A without inhibitors

Roche said Tuesday that the FDA granted breakthrough therapy designation to Hemlibra (emicizumab-kxwh) for people with haemophilia A without factor VIII inhibitors. The therapy was approved in the US last year for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adults and children with haemophilia A with factor VIII inhibitors.

"Hemlibra is the first medicine to show superior efficacy compared to factor VIII prophylaxis, the standard of care for people with haemophilia A without inhibitors, in an intra-patient comparison," remarked Sandra Horning, head of global product development at Roche. The company noted that the FDA's decision is based on data from the Phase III HAVEN 3 study, which showed that Hemlibra prophylaxis demonstrated a significant and clinically meaningful reduction in treated bleeds compared to no prophylaxis in people 12 years or older with haemophilia A without inhibitors.

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In February, Hemlibra was authorised by the European Commission for the treatment of bleeding episodes in patients with haemophilia A with factor VIII inhibitors, gaining clearance in Japan the next month in the same indication.

However, Roche's Genentech also recently confirmed that five deaths have occurred in patients receiving Hemlibra, with two of the deaths this year. The company noted that despite having "limited information" surrounding the circumstances, the deaths have all been deemed unrelated to Hemlibra by the investigator or treating physician.

For related analysis, see ViewPoints: Investors willing to overlook Hemlibra deaths – for now.

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