Production & Manufacturing
Process & Production

Roche secures priority review from FDA for haemophilia A drug emicizumab

PBR Staff Writer Published 25 August 2017

Roche's emicizumab prophylaxis (preventative) has been granted priority review by the US Food and Drug Administration (FDA) for haemophilia A with factor VIII inhibitors.

The bispecific monoclonal antibody is intended to be used as a once-weekly subcutaneous treatment for the disease to cover adults, adolescents and children.

According to Roche, emicizumab binds the IXa and X factors which are proteins that are needed to activate the natural coagulation cascade and repair the blood clotting process.

Roche stated that Biologics License Application (BLA) for emicizumab is based on results from two phase 3 studies HAVEN 1 and HAVEN 2.

The US regulator is likely to make a decision on emicizumab’s approval by 23 February 2018.

Roche chief medical officer and global product development head Sandra Horning said: “Roche has a history of developing innovative antibody therapies to address some of the highest unmet medical needs.

“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.”

Horning concluded that the company is working with the FDA to try and introduce the new prophylactic treatment option to haemophilia A inhibitor patients as early as possible.

Roche is also looking for approval of emicizumab in Europe where it has submitted data collected from the HAVEN 1 and HAVEN 2 to the European Medicines Agency (EMA). The haemophilia A drug will be reviewed by the European regulator under accelerated assessment.

Emicizumab is undergoing additional studies to assess its performance in patients having haemophilia A both with and without inhibitors. Likewise, less frequent dosing regimens of the drug are also being evaluated by the Swiss pharma company.

Image: Site Roche Basel. Photo: courtesy of F. Hoffmann-La Roche Ltd.