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US FDA lifts clinical hold on Sanofi, Alnylam's hemophilia trial

The U.S. Food and Drug Administration lifted the hold on clinical studies for Sanofi and Alnylam Pharmaceuticals Inc.'s hemophilia treatment fitusiran, allowing research to continue after it was paused due to safety concerns following a patient death.

Alnylam, which is developing fitusiran with Paris-based Sanofi, will now continue with clinical studies of the medicine to treat patients with hemophilia A and B. The studies include the phase 2 open-label extension study in which a patient died and the Atlas phase 3 program.

Previously, Alnylam and the FDA had reached alignment on new clinical risk mitigation measures, including protocol-specified guidelines and additional investigator and patient education concerning reduced doses of replacement factor or bypassing agent to treat any breakthrough bleeds in fitusiran studies.

The FDA has now approved the protocol amendments and other updated clinical materials for fitusiran studies.

Roche Holdings AG's Hemlibra, a monoclonal antibody also known as ACE910, was the first new medicine to be approved to treat hemophilia in 20 years. But ACE910 also suffered from safety concerns after patient deaths in clinical trials, and the approval on Nov. 17 included a so-called 'black box' warning highlighting potential severe blood clots from the weekly under-the-skin injection. Others competing in the highly competitive field include Novo Nordisk A/S and Shire plc.

At an analyst meeting in Paris Dec. 13, Sanofi's head of rare diseases Rand Sutherland said discussions with the U.S. regulator were ongoing and he anticipated resuming the clinical trials "imminently." Fitusiran, a protein targeted anti-thrombin, can treat both Hemophilia A and B, as well as patients with or without inhibitors. "We're actually quite excited about this," Sutherland said.

Sanofi's meeting came just after the American Society of Hematology meeting held in Atlanta, at which exciting data was presented by several companies.

"It's a tremendous time in Hemophilia," Sutherland said with regard to the ASH data. "This is a very dynamic and evolving landscape, I don't think the dye is fully cast yet."