India has approved Qfitlia (Fitusiran), a new treatment designed to prevent or reduce bleeding episodes in people living with hemophilia A or B, marking a significant expansion of preventive treatment options for patients aged 12 years and older.

 

The Central Drugs Standard Control Organization (CDSCO), under the Drug Controller General of India (DCGI), has granted marketing authorization for the therapy, the first antithrombin-lowering treatment approved for routine prophylaxis in this patient population, including those with or without factor VIII or IX inhibitors.

 

The approval is based on findings from the ATLAS Phase 3 clinical development programme, which demonstrated clinically meaningful protection against bleeding, measured through annualized bleeding rates (ABR), among people living with hemophilia, both with and without inhibitors.

 

The development comes as India continues to face a substantial hemophilia burden, with an estimated 28,900 registered people living with the condition. For patients who rely on on-demand treatment, bleeding episodes can mean repeated hospital visits, joint complications and disruptions to everyday life.

 

Parwas Mahajan, Business Head, Rare Diseases, Sanofi India, said the approval could help expand access to preventive care.

 

"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."

 

He added: "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."

 

Unlike conventional approaches that replace the clotting factors missing in people with hemophilia, Fitusiran works by lowering antithrombin, a natural inhibitor of blood clot formation. The approach aims to rebalance the body's coagulation system and support bleeding prevention in people with hemophilia A or B, including those with inhibitors.

 

Dr. Milan Choksey, Medical Lead, Rare Diseases Business, Sanofi India, highlighted the scientific shift behind the treatment.

 

"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 emphasis on proactive antithrombin monitoring underscores the importance of appropriate clinical oversight as this treatment approach enters India's hemophilia care landscape.