FDA grants priority review to Roche's filing for Hemlibra for haemophilia A without factor VIII inhibitors

Roche's Genentech unit announced Tuesday that the FDA granted priority review to a filing seeking clearance of Hemlibra (emicizumab-kxwh) for adults and children with haemophilia A without factor VIII inhibitors. The company noted that the submission, which is based on data from the Phase III HAVEN 3 study, has a target review date of October 4.

Hemlibra gained approval 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. Meanwhile, earlier this year, the FDA granted breakthrough therapy designation to the drug for people with haemophilia A without factor VIII inhibitors.

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In the HAVEN 3 study, adults and adolescents aged 12 years or older with haemophilia A without factor VIII inhibitors who received Hemlibra prophylaxis every week or every two weeks showed a 96 percent and 97 percent reduction in treated bleeds, respectively, compared to those who received no prophylaxis. Last month, Roche and partner Chugai Pharmaceutical also indicated that in a subset of patients in the trial who previously received factor VIII prophylaxis, Hemlibra reduced bleeds by 68 percent compared to prior therapy.

Results from the HAVEN 3 study have also been submitted to regulators in Europe, where Hemlibra was recently approved for routine prophylaxis of bleeding episodes in people with haemophilia A with factor VIII inhibitors. For related analysis, read KOL Views Results: Leading haematologist thinks HAVEN 3 data will likely speed adoption of Hemlibra in non-inhibitor patients, and see Physician Views Results: Evidence continues to accumulate for Hemlibra's long-term play in haemophilia A.

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