On 21 October 2026, the Central Drugs Standard Control Organisation (CDSCO) granted marketing authorisation in India for Qfitlia, whose active ingredient is fitusiran. The therapy is approved for routine prophylaxis to prevent bleeding episodes in adults and adolescents aged 12 years and older with haemophilia A or B, with or without factor VIII or IX inhibitors.
Haemophilia and Clotting Factors
Haemophilia is an inherited bleeding disorder in which deficiency or dysfunction of a blood-clotting factor can cause prolonged bleeding. Haemophilia A is associated with deficiency of factor VIII, while haemophilia B is associated with deficiency of factor IX. Inhibitors are antibodies that can develop against replacement clotting factors and reduce their effectiveness. The Qfitlia authorisation covers patients both with and without these inhibitors. The therapy is intended for prophylaxis, which means treatment given regularly to prevent bleeding rather than only after a bleed occurs.
Fitusiran and Antithrombin
Fitusiran is an antithrombin-lowering therapy. Antithrombin is a natural anticoagulant protein that limits the activity of clotting enzymes, including thrombin and factor Xa. Lowering antithrombin can increase thrombin generation and help restore haemostatic balance in people with haemophilia. Qfitlia is administered by injection, with a regimen requiring as few as six injections annually. Its Indian approval was based on the ATLAS phase 3 clinical development programme, which demonstrated clinically meaningful bleed protection.
Prophylaxis and Drug Regulation
Routine prophylaxis is preventive treatment used to reduce the frequency of bleeding episodes and related complications. Qfitlia is the first antithrombin-lowering therapy approved in India for routine prophylaxis in haemophilia. The CDSCO is India’s national drug regulatory authority under the Ministry of Health and Family Welfare. It regulates the import, manufacture, distribution and sale of drugs, and the Drugs Controller General of India heads the organisation.
Important Facts for Exams
- Haemophilia A involves factor VIII, while haemophilia B involves factor IX.
- Antithrombin is a natural inhibitor of thrombin and factor Xa.
- Fitusiran is the active ingredient in Qfitlia.
- The CDSCO operates under the Ministry of Health and Family Welfare.
Qfitlia is authorised for patients aged 12 years and older with haemophilia A or B, including those with factor inhibitors. The approved therapy is used for prevention of bleeding episodes rather than treatment of an acute bleed.
