Death in trial of Roche’s potential blockbuster haemophilia drug

pharmafile | February 24, 2017 | News story | Medical Communications, Research and Development Novo Nordisk, Roche, Shire, haemophilia 

Roche has reported a death in the trial of its haemophilia treatment, emicizumab, that will only add to safety concerns over the drug. It had previously reported four serious adverse events (SAE), involving blood clotting, in Phase 3 trials but these had been resolved with  two patients returning to use the treatment.

The latest news of a death in the trial is certain to reawaken fears regarding safety. Roche issued a statement to the European Haemophilia Consortium regarding the death, releasing that the patient had suffered from a serious rectal haemorrhage before receiving bypassing agents, the patient then developed Thrombotic Microangiopathy (TMA). The patient died as a result of the SAEs.

Roche was careful to note that the patient had refused blood transfusions as a factor in their death. It also noted that the death was as a result of the rectal haemorrhage that is not related to treatment with emicizumab.

“The preliminary assessment is that the clinical and laboratory characteristics of this case of TMA are consistent with what was observed in the two previously reported cases; however, our evaluation of the available information is ongoing”, Roche released as part of the statement.

The two previously reported cases refers to the previous Phase 3 trial, where patients who had experienced TMA were treated with bypassing agents – known to increase the risk factor of thrombosis.

The news will come as a blow to Roche but a boon to Novo Nordisk and Shire – the latter two being the dominant companies in the haemophilia market. Novo Nordisk’s NovoSeven had sales of $1.4 billion in 2016 while Shire’s haematology products, though experiencing slow growth, are equally important to its portfolio. Both are expected to slip should Roche bring emicizumab to market, with analysts expecting the drug to reach blockbuster status.

Ben Hargreaves

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