FDA Approves Roche’s Hemophilia A Biologic and Expands Indication for Leukemia Drug

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The agency has approved a biologic-based new molecular entity for treating hemophilia A and has expanded the indication for a leukemia drug to now treat a common form of non-Hodgkin lymphoma.

FDA has approved Hemlibra (emicizumab-kxwh), a biologic-based new molecular entity for treating hemophilia A, and has approved an additional indication for Gazyva (obinutuzumab), a leukemia biologic, to now treat follicular lymphoma. Both biologics are products by Roche.

The Nov. 16, 2017 approval of Hemlibra is specifically for preventing or reducing the frequency of bleeding episodes associated with hemophilia A in adult and pediatric patients who have developed Factor VIII (FVIII) inhibitors.

FDA granted the Hemlibra application priority review and breakthrough therapy designations. The biologic also received orphan drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases.

“Today’s approval of Hemlibra represents an important advancement for people with hemophilia A with inhibitors, who have struggled to manage their bleeding disorder and haven’t had a new medicine in nearly 20 years,” said Sandra Horning, MD, Roche’s chief medical officer and head of global product development, in a company press release. “We believe Hemlibra will improve protection against bleeds and reduce the treatment administration burden for people with hemophilia A with inhibitors, and we are committed to helping them access this medicine.”

The biologic is also being reviewed under accelerated assessment by the European Medicines Agency, and submissions to health authorities around the world are ongoing. It is a bispecific factor IXa- and factor X-directed antibody designed to bring together the factor IXa and factor X proteins that are required to activate the natural coagulation cascade and restore the blood clotting process that is lacking due to hemophilia A. The biologic was created by Japanese pharmaceutical company Chugai Pharmaceutical and is being co-developed by Chugai, Roche, and Genentech, a Roche company.


The second approval, the expanded indication for Gazyva, was granted by FDA on Nov. 17, 2017, and that biologic is now approved in combination with chemotherapy for treating previously untreated advanced follicular lymphoma (stage II bulky, III, or IV), the most common form of non-Hodgkin lymphoma. The biologic is an engineered monoclonal antibody designed to target CD20, which is a protein expressed on certain B cells, but not on stem cells or plasma cells.

Gazyva is marketed as Gazyvaro in the European Union and Switzerland. It is currently approved in more than 80 countries in combination with chlorambucil for treating previously untreated chronic lymphocytic leukemia, in combination with bendamustine for treating certain types of previously treated follicular lymphoma, and in combination with chemotherapy for previously untreated, follicular lymphoma. In addition, combination studies are underway investigating its use with other approved or investigational medicines, including cancer immunotherapies and small-molecule inhibitors, across a range of blood cancers.

Source: FDA and Roche