Roche receives FDA Priority Review for drug with potted history

Roche, alongside its subsidiary, Genentech, announced that it has received Priority Review for its haemophilia A drug, emicizumab, and that the agency has accepted its BLA. The application process will be watched with some interest after the drug has already caused quite a stir before even reaching the market.

The product could potentially become a blockbuster treatment in the area, to rival Novo Nordisk and Shire’s presence in the market. The BLA was granted on the basis of Phase 3 results, with interim results showing that after a median observation time of 12 weeks, only one of 19 children reported a treated bleed. As well as this, there were no reported joint or muscle bleeds.

"Genentech has a history of developing innovative antibody therapies to address some of the highest unmet medical needs," said Sandra Horning, Chief Medical Officer and Head of Global Product Development. "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. 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 strong results from the trial, however, have been marred by serious incidents in trials. It was revealed earlier in the year that a patient had died after suffering two serious adverse events. This came after several previously reported incidents of thrombotic microangiopathy.

The situation was muddied further as Shire alleged that Roche’s explanation of the death were "incomplete and misleading statements", regarding the use of its aPCC medications alongside emicizumab. The suggestion being that Roche had attempted to pass the buck on the death to its own product, Feiba. Shire received an injunction from a court in Hamburg, Germany, relating to the statements from Roche.