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Roche wins CHMP backing for Hemlibra

Haemophilia A treatment on course for European approval


European regulatory advisors have given their backing to Roche’s Hemlibra (emicizumab), putting the haemophilia A treatment on course for European approval later this year.

Roche is seeking a licence for the drug to be used to treat routine prophylaxis of bleeding episodes in haemophilia A patients with factor VIII inhibitors.

The Committee of Human Medicinal Products (CHMP)’s decision on the once-weekly subcutaneous treatment covers its use for all age groups, and Roche is hoping the drug may reduce the disease burden of managing the disease.

Sandra Horning, chief medical officer for Roche, said: “Hemlibra has been shown to effectively reduce the frequency of bleeds compared to currently available medicines, and with once-weekly administration by injection under the skin, it could also greatly reduce the treatment administration burden, particularly for young children with haemophilia A with inhibitors and their families.”

The CHMP’s recommendation is based on the results of two phase III clinical studies for people with this particular disease indication.

The HAVEN 1 trial - which studied the drug in patients 12 years and older - showed a statistically significant reduction in treated bleeds of 87% compared to no prophylaxis.

Meanwhile interim results from the HAVEN 2 study, which saw the drug treating children younger than 12 years of age, revealed that 87% of children who received Hemlibra prophylaxis experienced no treated bleeds.

Nearly one in three people with severe haemophilia A can develop inhibitors to factor VIII replacement therapies, according to the European Haemophilia Consortium, putting them at greater risk of life-threatening bleeds or repeated bleeding episodes that can cause long-term joint damage.

Horning added: “The positive CHMP opinion represents a significant step towards bringing this new treatment to people with haemophilia A with inhibitors in Europe.”

CHMP decisions are usually translated into full approval from the European Commission within three months. If Hemlibra is passed it will be the first new medicine to treat haemophilia A with inhibitors in Europe in over 20 years.

Meanwhile in the US, the drug was recently licenced for use in the same indication by the Food and Drug Administration (FDA) three months earlier than expected based on positive data.

Article by
Gemma Jones

30th January 2018

From: Regulatory



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