CDSCO approves Sanofi’s Qfitlia for haemophilia A and B in India

The Central Drugs Standard Control Organization (CDSCO), under the Drug Controller General of India (DCGI), has granted marketing authorisation to Qfitlia (fitusiran), an antithrombin-lowering (AT) therapy for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adults and adolescents aged 12 years or older living with haemophilia A or B, with or without factor VIII or IX inhibitors.

The approval is based on data from the ATLAS Phase III clinical development programme, which demonstrated clinically meaningful bleed protection, as measured by annualised bleeding rates (ABR), among people living with haemophilia, with and without inhibitors.

Parwas Mahajan, Business Head, Rare Diseases, Sanofi India, said, “India carries a heavy Hemophilia burden – an estimated ~28.9k# registered people living with Hemophilia. Of those receiving treatment, a significant percent is still on on-demand therapy, meaning they treat bleeds only after they occur, living in constant anticipation of the next joint bleed, the next hospital visit, the next disruption to their lives and livelihoods. The approval expands prophylactic treatment options for people living with Hemophilia in India and (Prophylaxis) is increasingly recognized as an important treatment approach in the management of hemophilia, and today’s approval of Qfitlia® is a significant step toward making that a reality. Our vision is a ‘Hemophilia-free mind’ and a life where people are not defined by their condition, where they are not held hostage by the fear of the next bleed. Qfitlia®, with its unique ability to protect both those living with Hemophilia A and Hemophilia B (with or without inhibitors) with as few as six injections a year, has the potential to significantly expand access to prophylaxis in India. Sanofi is deeply committed to India’s Hemophilia community, and this approval marks the beginning of a long-term partnership with the objective of supporting improved awareness and access to appropriate hemophilia care in India.”

Dr Milan Choksey, Medical Lead, Rare Diseases Business, Sanofi India, said, “The approval of Qfitlia introduces a concept that is still largely new to India: the role of antithrombin in hemostasis, and the power of lowering it to rebalance the coagulation system. For decades, our approach to Hemophilia has been to replace what is missing (factor VIII or Factor IX). Fitusiran asks a fundamentally different question: what if, instead of replacing the missing piece, we removed a brake on the system?

“Antithrombin is a natural inhibitor of thrombin generation. By reducing AT levels, Fitusiran allows the body’s remaining coagulation machinery to work more effectively, regardless of whether the person has Hemophilia A or B, with or without inhibitors. This is the science of rebalancing and introduces a novel treatment approach that may be of interest to treating physicians. Equally important is the role of proactive AT monitoring.

“The dosing of Qfitlia is guided by AT levels – a measurable, reliable biomarker that allows physicians to personalize treatment and optimise the risk-benefit profile for each person living with Hemophilia. This is a new discipline for India, and Sanofi is committed to supporting education and appropriate implementation of AT monitoring, because the science of Fitusiran is only as powerful as the ecosystem that supports it.”