CDSCO panel Approves Sanofi's Protocol Amendment Proposal for to study Fitusiran

New Delhi: The Subject Expert Committee (SEC) functional under the Central Drug Standard Control Organisation (CDSCO) has approved drug major Sanofi's protocol amendment proposal of Fitusiran, a subcutaneous investigational small interfering RNA therapeutic, targets antithrombin to rebalance haemostasis in people with haemophilia A or haemophilia B, irrespective of inhibitor status.

However, this approval is subject to the condition that the firm has to submit the that interim analysis reports every 24 weeks of the proposed drug.

This came after the firm presented protocol amendment 03 version 1 dated 20.10.2023 and protocol amendment 04 version 1 dated 24.11.2023 protocol No. EFC1546.

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

The study medication, fitusiran, employs a unique mechanism of action—small interfering with RNA, or siRNA. The siRNA can be customized to block 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 21st May 024, the expert panel reviewed the protocol amendment 03 version 1 dated 20.10.2023 and protocol amendment 04 version 1 dated 24.11.2023 protocol No. EFC1546.

After detailed deliberation, the committee recommended the approval of protocol amendment as presented by the firm subject to the condition that interim analysis reports every 24 weeks shall be submitted to CDSCO.

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