FDA Grants Priority Review to Genentech's Emicizumab

Genentech, a member of Roche Group, announced on Wednesday that the FDA has accepted its Biologics License Application (BLA) and granted priority review for emicizumab, a once-weekly subcutaneous prophylaxis for adults, adolescents, and children with hemophilia A who have factor VIII inhibitors. A regulatory decision on the drug is expected by February 23, 2018.

Nearly 1 in 3 patients with hemophilia A, an inherited clotting disorder that affects approximately 20,000 people in the United States, develop inhibitors to standard factor VIII replacement therapies. Development of these inhibitors limits patients’ treatment options and increases the risk of serious bleeds. Emicizumab, an investigational bispecific monoclonal antibody, is designed to bring together factors IXa and X, which are required to activate the natural coagulation cascade and restore the blood-clotting process.

“Genentech has a history of developing innovative antibody therapies to address some of the highest unmet medical needs,” said Sandra Horning, MD, CMO and head of global product development at Genentech. “Results of our [phase 3] study in adults and adolescents as well as early [phase 3] results in children showed that emicizumab has significant potential to help people with hemophilia A with inhibitors, who face major challenges in preventing and treating bleeds.”

Genentech’s BLA for emicizumab is based on the results of the phase 3 HAVEN 1 and HAVEN 2 studies. In the HAVEN 1 study, 62.9% of patients receiving emicizumab experienced zero treated bleeds (compared with 5.6% of patients receiving on-demand bypassing agents) after 31 weeks of emicizumab prophylaxis. Interim results from the single-arm HAVEN 2 study in children under 12 years showed that 1 patient (of 19) receiving emicizumab reported a treated bleed at a median observation time of 12 weeks.

Some analysts suggest that if emicizumab is approved, the novel biologic could generate approximately $1.5 billion in sales for Genentech (and its parent company, Roche) by 2022. Those earnings could offset the potential erosion of its earnings from Roche’s cancer drugs, bevacizumab (Avastin), trastuzumab (Herceptin), and rituximab (Mabthera, Rituxan), which together brought in $22.1 billion in sales in 2016. All 3 blockbuster drugs face oncoming biosimilar competition.

Genentech and Roche’s development of novel biologics also includes other drugs with the potential to earn strong sales numbers. A few other monoclonal antibodies being developed by the 2 companies are currently in phase 3 development, including obinutuzumab, for the treatment of non-Hodgkin’s lymphoma; etrolizumab, for the treatment of ulcerative colitis and Crohn’s disease; gantenerumab, for the treatment of Alzheimer’s disease; and ocrelizumab, for the treatment of multiple sclerosis.