Roche’s emicizumab’s Phase III trial in haemophilia A has most experts questioning drug overall safety profile, clinical future unclear

07 Mar 2017

Roche’s (VTX:ROG) report of a patient death in its Phase III ACE910 (emicizumab) HAVEN 1 study in haemophilia A had experts question a trial investigator’s claim the death occurred independently of the drug.

Although two analyst reports also note that the death was unlikely emicizumab associated, three experts argued that emicizumab’s thrombotic microangiopathy (TMA) effects likely prevented the patient from receiving sufficiently aggressive therapy to stop rectal bleeding, resulting in his death. TMA and major rectal bleeds don’t typically occur in haemophilia A, which supports emicizumab holding responsibility, one expert added. A fourth expert added, however, the patient death can’t be accurately assessed without knowing the bypassing agent and its dosing.

Even if the death hadn’t occurred, emicizumab’s TMA incidence is unacceptable, two experts said. A separate expert noted, however, Roche previously reported that both TMA cases in the trial resolved, with one patient restarting emicizumab.

Certain experts also commented on a second patient death from an intracranial haemorrhage whilst receiving emicizumab.

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