FDA clears Roche's haemophilia A therapy Hemlibra

The FDA on Thursday announced the approval of Roche's Hemlibra (emicizumab-kxwh) to prevent or reduce the frequency of bleeding episodes in patients with haemophilia A who have developed Factor VIII inhibitors. Richard Pazdur, acting director of the Office of Hematology and Oncology Products in the FDA's Center for Drug Evaluation and Research, said the "approval provides a new preventative treatment that has been shown to significantly reduce the number of bleeding episodes." 

Roche noted that approval of the drug was backed by efficacy and safety data from the Phase III HAVEN 1 and HAVEN 2 studies. Results from the HAVEN 1 study, which were released in June, showed that Hemlibra was associated with an 87-percent decline in treated bleeds in patients aged 12 and older with haemophilia A with inhibitors versus patients who did not receive prophylactic treatment, as well as a 79-percent reduction in treated bleeds compared to previous treatment with bypassing agent prophylaxis. Meanwhile, in the HAVEN 2 trial of children younger than 12, interim results revealed that 87 percent of patients with haemophilia A and inhibitors who were treated with Hemlibra prophylaxis experienced no treated bleeds. 

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Chief medical officer Sandra Horning remarked "today’s approval of Hemlibra represents an important advancement for people with haemophilia A with inhibitors, who have struggled to manage their bleeding disorder and haven’t had a new medicine in nearly 20 years.” 

Roche, which expects the therapy to cost approximately $482 000 during the first year of treatment and about $448 000 the following year, said that it will launch Hemlibra in the US shortly. The once-weekly subcutaneous therapy was previously granted priority review and breakthrough therapy designation by the FDA, while the drug has also been awarded orphan drug status by the agency. 

The FDA however indicated that the drug's label will contain a black boxed warning that thrombotic microangiopathy and thromboembolism have been observed in patients who were also given an activated prothrombin complex concentrate to treat bleeds for 24 hours or more while taking Hemlibra. (For related analysis, read ViewPoints: Shire’s joy over label for Roche’s Hemlibra may be short-lived. See also KOL Views: Roche’s Hemlibra approved with a black box – consequential or trivial?)

Meanwhile, the drugmaker indicated that results from HAVEN 1 and HAVEN 2 studies are being reviewed under accelerated assessment by the EMA, while submissions to health regulators around the world, including Japan, are ongoing. Hemlibra was previously given orphan drug designation by regulators in Japan, where it is marketed by partner Chugai Pharmaceutical. 

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