Alnylam announces lift of FDA clinical hold on Fitusiran

Alnylam Pharmaceuticals (ALNY) and Sanofi Genzyme, the specialty care global business unit of Sanofi (SNY), announced today that the U.S. Food and Drug Administration has lifted the hold on clinical studies with fitusiran, including the Phase 2 open-label extension study and the ATLAS Phase 3 program.

Alnylam and the FDA had previously 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.

Fitusiran is an investigational RNAi therapeutic targeting antithrombin for the treatment of patients with hemophilia A and B.

It is designed to lower levels of AT with the goal of promoting sufficient thrombin generation to restore hemostasis and prevent bleeding.

Alnylam suspended patient dosing in all ongoing studies of its RNA therapy fitusiran to treat hemophilia A or B, after a patient died of swelling in the brain in a Phase 2 open-label extension trial.

Fitusiran, also known as ALN-AT3SC, is designed to specifically lower levels of antithrombin — a protein that inhibits clotting — to improve production of thrombin, a clotting factor. As such, it aims to create a better balance of clotting components that would prevent bleeding events in hemophilia A and B patients.

Alnylam and the FDA had previously 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.

ALNY closed at $123.66. It last traded at $128.10. SNY closed at $43.09.

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