Roche's Hemlibra gains expanded EU approval to include patients with haemophilia A without factor VIII inhibitors

The European Commission approved Roche's Hemlibra (emicizumab) for routine prophylaxis of bleeding episodes in people with severe haemophilia A without factor VIII inhibitors, the company reported Thursday. Sandra Horning, head of global product development, said "today's approval is a landmark moment as Hemlibra is the first new class of treatment for people with severe haemophilia A without inhibitors in nearly 20 years."

The approval was based on findings from the pivotal HAVEN 3 and HAVEN 4 studies. In the HAVEN 3 trial, in people with haemophilia A without factor VIII inhibitors, Hemlibra prophylaxis led to significant and clinically meaningful reductions in treated bleeds compared to no prophylaxis, and compared to prior treatment with factor VIII prophylaxis in a prospective intra-patient comparison. Meanwhile, in the HAVEN 4 study in people with haemophilia A with and without factor VIII inhibitors, Hemlibra showed a clinically meaningful control of bleeding when dosed every four weeks.

Hemlibra initially gained EU clearance last year for the treatment of bleeding episodes in patients with haemophilia A with factor VIII inhibitors. Roche noted that following the latest approval, Hemlibra can now be used in all age groups, and can also now be used at multiple dosing options, including once weekly, every two weeks or every four weeks, for all indicated people with haemophilia A, including those with factor VIII inhibitors.

Meanwhile, the FDA expanded clearance of Hemlibra in October last year for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adults and children with haemophilia A without factor VIII inhibitors. The therapy, which was created by Chugai Pharmaceutical and is being co-developed with Roche, generated sales of 111 million Swiss francs ($111 million) in the fourth quarter of 2018.

Last month, Roche entered an agreement to buy gene therapy developer Spark Therapeutics for approximately $4.3 billion, gaining the latter's haemophilia A programme SPK-8011, which is expected to start Phase III testing this year. Spark's other gene therapy programmes include SPK-8016 in Phase I/II development targeting the haemophilia A inhibitor population.

For related analysis, read ViewPoints: Roche pivots into gene therapy.

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