CDSCO Panel Approves Sanofi's Protocol Amendment proposal for Fitusiran study

New Delhi: The Subject Expert Committee (SEC) functional under the Central Drug Standard Control Organisation (CDSCO) has approved the drug major Sanofi's protocol amendment proposal of the clinical trial titled "A Phase 3, single-arm, multicenter, multinational, open label,one-way crossover study to investigate the efficacy and safety of fitusiran prophylaxis in male participants aged ≥ 12 years with severe hemophilia A or B, with or without inhibitory antibodies to factor VIII or IX"

This came after the firm presented protocol amendment number 03, version 01 dated 30.01.2023 protocol No. EFC17574. This is a multicenter, multinational, open-label, one-way cross-over, Phase 3, single-arm study for treatment of hemophilia.

The purpose of this study is to measure the frequency of treated bleeding episodes with fitusiran in male adult and adolescent (≥12 years old) participants with hemophilia A or B, with or without inhibitory antibodies to factor VIII or IX who have switched from their prior standard of care treatment.

Fitusiran is a monthly subcutaneous RNA interference therapy targeting antithrombin to increase thrombin generation and reduce bleeding in hemophilia with or without inhibitors.

Fitusiran is a small interfering RNA or siRNA. The siRNA can be customized to block the production of any protein, leading to lower levels of the target protein. In this case, it blocks antithrombin, which inhibits a coagulation protein called thrombin.

At the recent SEC meeting for Hematology held on 8th August 2024, the expert panel reviewed the protocol amendment number 03, version 01 dated 30.01.2023 protocol No. EFC17574.

After detailed deliberation, the committee recommended the approval of the protocol amendment as presented by the firm.

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