Genentech’s Hemophilia A Drug Gets Breakthrough Designation in Potential $11 Billion Market

Genentech’s Hemophilia A Drug Gets Breakthrough Designation in Potential $11 Billion Market
September 4, 2015
By Mark Terry, BioSpace.com Breaking News Staff

The U.S. Food and Drug Administration (FDA) today announced that it had given breakthrough therapy designation to Genentech for its hemophilia A with factor VIII inhibitors drug, ACE910.

The drug was part of a Phase I study in individuals with severe hemophilia A, a disease that prevents blood from clotting, which can lead to dangerous bleeding. ACE910 is a humanized bispecific monoclonal antibody that simultaneously binds factors IXa and X, and promotes blood coagulation. It is administered subcutaneously once a week.

The drug was invented by Chugai Pharmaceutical Co., Ltd. and is being co-developed by Genentech, part of the Roche Group. Genentech is planning to start a Phase III trial of ACE910 by the end of this year in patients with hemophilia A with factor VIII inhibitors, and in patients without inhibitors in 2016. The company also expects to initiate a trial in pediatric patients with hemophilia A in 2016.

“People with hemophilia A may require regular and frequent infusions of replacement clotting factor to reduce the risk of dangerous bleeding, and they can develop inhibitors that make replacement ineffective,” said Sandra Horning, chief medical officer and head of Roche ’s Global Product Development in a statement. “We are pleased that the FDA has granted breakthrough therapy designation for ACE910, recognizing an unmet need for patients with inhibitors and the promise of these early data. Genentech has been developing antibody treatments for people with blood disorders for over 20 years, and we are excited to expedite the development of a potential new treatment for hemophilia A.”

Breakthrough therapy designation accelerates the development and review processes for certain drugs that show early clinical data of significant improvement over current treatment options.

If approved, ACE910 would compete with treatments offered by Novo Nordisk and Baxalta. Baxalta currently markets Advate, Bebulin, Feiba NF, Hemofil M Antihemophilic Factor, Recombinate, and Rixubis for various forms of hemophilia. On July 17, 2015, Baxalta announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) had come down on the positive side for a marketing authorization for Obizur for the treatment of bleeding episodes in adults with hemophilia caused by antibodies to Factor VIII. The drug is approved in the U.S. and is being reviewed by regulators in Canada, Switzerland, Australia and Columbia.

Novo Nordisk markets NovoSeven RT, Novoeight, Tretten, and Norditropin for several types of hemophilia.

Novo Nordisk has been in conflict with Baxter International , which spun off Baxalta in July of this year, over patient claims over the hemophilia treatment Novoeight. Baxter claimed Novoeight infringed some of its patents, which led to an investigation by the U.S. International Trade Commission. Baxter argues that the drug was manufactured using processes developed by Baxter and are patent protected.

The market for hemophilia drugs is projected to hit $11 billion in 2016.

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