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Roche wins FDA priority review for haemophilia A drug

Emicizumab prophylaxis could be approved early next year


The US Food and Drug Administration (FDA) has awarded priority review status to Roche’s emicizumab prophylaxis, a once weekly subcutaneous treatment used in conjunction with factor VIII inhibitors for patients with haemophilia A.

The decision follows the phase III HAVEN 1 study in adults and adolescents, as well as interim results from the phase III HAVEN 2 study in children younger than 12 years of age.

Sandra Horning, Roche’s chief medical officer, said: “Results of our phase III study in adults and adolescents as well as early phase III results in children showed that emicizumab has significant potential to help people with haemophilia A with inhibitors, who face major challenges in preventing and treating bleeds.”

Haemophilia affects around 320,000 people worldwide, with 50-60% of that number suffering from a ‘severe’ form of the disorder.

Horning added: “We are working with the FDA to hopefully bring this new prophylactic treatment option to the haemophilia A inhibitor community as soon as possible.”

The FDA is expected to make a decision on the pharma giant’s injection late February next year.

Meanwhile, Roche has additional trials of the drug ongoing, with and without inhibitors, to explore other, less frequent dosing regimens.

Article by
Gemma Jones

29th August 2017

From: Regulatory



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