Roche’s emicizumab has largely uncertain haemophilia A approval forecasts after undetailed Phase III safety read outs – experts

08 Feb 2017

Despite positive efficacy results, Roche’s (VTX:ROG) ACE910’s (emicizumab) Phase III outcomes lack sufficient detail to gauge the drug’s safety profile and subsequent approval prospects in haemophilia A, three experts agreed.

The recently released trial information raises certain safety alarms, with details including the patient discontinuation rate and why some patients didn’t restart therapy after suffering toxicities missing, one expert noted. A separate expert added emicizumab’s troubling safety outcomes may have arisen due to baseline patient risk factors or concurrent therapy, adding further information is required. The Phase III’s relatively small size also doesn’t boost safety confidence, a third expert added.

Yet, a fourth expert argued, emicizumab’s approval prospects shouldn’t be affected by its Phase III safety outcomes as thrombosis is anticipated given the drug’s mechanism of action (MOA) and relatively high dosing. Trial success also suggests emicizumab’s benefits outweigh its toxicities, encouraging approval, she added. Experts spoke on the sidelines of the European Association of Haemophilia and Allied Disorders in Paris.

An analyst report anticipates peak sales could reach more than CHF 1bn (USD 1bn). A separate report notes HAVEN 1’s success but adds emicizumab’s safety confidence is lacking.

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