NEW DELHI: People with haemophilia A and B in India may soon have another option to prevent bleeding episodes, with the drug regulator approving Sanofi’s Qfitlia (fitusiran) for patients aged 12 years and above, including those who have developed inhibitors against conventional treatment.
The Central Drugs Standard Control Organisation (CDSCO) has approved the drug for routine preventive treatment, which aims to reduce the frequency of bleeding rather than treat it only after it occurs, said Sanofi in a statement here. The approval covers patients with and without inhibitors, a complication that can make standard factor replacement less effective.
The drug could expand preventive treatment options for India’s estimated 1.4 lakh people living with haemophilia, according to an estimate cited by Sanofi from the Indian Council of Medical Research (ICMR). However, the Hemophilia Federation of India’s 2023-24 annual report lists around 28,900 registered patients, highlighting the gap between estimated disease burden and documented cases.
Haemophilia is an inherited bleeding disorder in which the blood does not clot properly. Haemophilia A results from a deficiency of clotting factor VIII, while haemophilia B is caused by a deficiency of factor IX. Repeated bleeding, particularly into joints, can cause chronic pain, joint damage and disability.
Unlike conventional treatment, which replaces the missing clotting factor, fitusiran works by lowering antithrombin, a protein that limits blood clotting. This helps restore the balance between the body’s clot-forming and clot-limiting mechanisms.
The drug is administered under the skin. Sanofi said its dosing regimen can require as few as six injections a year, potentially reducing the treatment burden for eligible patients.
The approval is based on the ATLAS Phase 3 clinical trial programme, which evaluated the drug in people with haemophilia A and B, with and without inhibitors.
A Phase 3 trial published in The Lancet in 2023 studied 57 people aged 12 years and above with haemophilia and inhibitors. The annualised bleeding rate was 1.7 among those receiving fitusiran, compared with 18.1 among those receiving on-demand treatment with bypassing agents. This represented a 90.8% reduction in the estimated bleeding rate. However, two participants receiving fitusiran experienced suspected or confirmed blood clots, underscoring the need for careful safety monitoring.
A 2025 analysis of the ATLAS-OLE study also reported sustained protection against bleeding with an antithrombin-based dosing regimen. The analysis found a 71% reduction in mean annualised bleeding rates compared with on-demand clotting factor concentrates. The researchers reported that some patients could maintain protection with six injections a year.
The treatment will require regular monitoring of antithrombin levels to guide dosing and reduce the risk of excessive clotting. International prescribing information also warns of potentially serious thrombotic events and gallbladder complications.
For India, the impact will depend not only on regulatory approval but also on affordability, availability, specialist care and access to the required monitoring facilities, said experts in the sector.
Parwas Mahajan, Business Head, Rare Diseases, Sanofi India, said the approval expands preventive treatment options for people with haemophilia. The company said it would also support awareness and education on appropriate use of the therapy.
The approval marks a new treatment option for eligible patients. Its wider public health benefit will depend on whether people who currently have limited access to preventive care can obtain the drug and receive the monitoring needed for safe treatment.
Ends
