Roche Hemophilia Drug Gets Breakthrough Therapy Designation

Roche Holding AG RHHBY member Genentech announced that the FDA has granted Breakthrough Therapy Designation to its hemophilia drug, Hemlibra (emicizumab-kxwh). The designation was granted to Hemlibra for treatment of people with hemophilia A without factor VIII inhibitors.

The designation aims to expedite the development and review of drugs intended to treat serious or life-threatening conditions and provide patients access to these as soon as possible.

This designation is based on data from the phase III HAVEN 3 study in people 12 years or older with hemophilia A without inhibitors. In the study, Hemlibra prophylaxis dosed subcutaneously every week or every two weeks showed a statistically significant and clinically meaningful reduction in treated bleeds compared to no prophylaxis. The study met its primary endpoint as well as key secondary endpoints with Hemlibra significantly reducing bleeding.

We note that the FDA approved Hemlibra for routine prophylaxis to event or reduce the frequency of bleeding episodes in adults and children with haemophilia A with factor VIII inhibitors in November 2017. Hemlibra was reviewed by the FDA under Priority Review and granted Breakthrough Therapy Designation in September 2015.

In January 2018, the EU Committee for Medicinal Products for Human Use ("CHMP") adopted a positive opinion for Hemlibra (emicizumab) for routine prophylaxis of bleeding episodes in patients suffering from hemophilia A with factor VIII inhibitors. The CHMP has recommended the drug for use in all age groups. The Marketing Authorisation Application is being reviewed under accelerated assessment.

A potential approval will further boost Roche's hematology portfolio which includes approved drugs like MabThera/Rituxan, Gazyva/Gazyvaro, Tecentriq and Venclexta 

We note that Roche is developing Venclexta in collaboration with AbbVie ABBV. Both companies are jointly commercializing the drug in the United States. AbbVie is commercializing the drug outside the United States.

Approval of new drugs and a potential label expansion of existing drugs bode well for Roche as its legacy drugs like Herceptin, MabThera are facing competition from biosimilars.

Novartis NVS has already launched its biosimilar version of Rituxan/ MabThera in Europe. Amgen AMGN has also obtained FDA approval for a biosimilar version of Avastin for treatment of five types of cancers including lung cancer, colorectal cancer, glioblastoma, renal cell carcinoma and cervix cancer.

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