Sanofi’s Qfitlia Gets India Nod For Bleeding Prevention In Haemophilia Patients; Experts Stress Access And Monitoring

For people living with haemophilia, a blood disorder, preventing recurrent bleeding and avoiding long-term joint damage remain major treatment goals. The Central Drugs Standard Control Organisation (CDSCO) approval of Sanofi’s Qfitlia (fitusiran) in India offers a new prophylactic treatment to prevent or reduce bleeds for patients aged 12 years and above with haemophilia A or B, with or without inhibitors. The French drug manufacturer announced the Indian marketing authorisation of the drug on October 21.

Doctors say its potential to reduce bleeding episodes and the need for frequent intravenous infusions could make treatment more convenient for patients. However, affordability, access and careful safety monitoring will be crucial to its wider use.

Haemophilia is an inherited bleeding disorder caused by a deficiency of clotting factor VIII in haemophilia A or factor IX in haemophilia B. Patients may experience prolonged or spontaneous bleeding, particularly into joints and muscles. Repeated joint bleeds can cause chronic pain, restricted movement and long-term disability.

Unlike conventional treatment, which replaces the missing clotting factor, fitusiran uses RNA interference (RNAi) technology to reduce the production of antithrombin, a natural protein that limits blood clotting. It targets the SERPINC1 gene, lowering antithrombin levels to rebalance the clotting system.

The approval follows the ATLAS Phase 3 clinical development programme, which evaluated fitusiran in patients with haemophilia A and B, including those with inhibitors.

A Phase 3 trial published in The Lancet in 2023 among patients with haemophilia and inhibitors found an estimated annualised bleeding rate of 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.

A separate Phase 3 trial, ATLAS-A/B, published in The Lancet Haematology in 2023, also found that preventive treatment significantly reduced bleeding in patients with severe haemophilia A or B without inhibitors compared with on-demand clotting-factor treatment.

The findings suggest that fitusiran could improve bleed control and reduce the burden of frequent infusions. However, individual outcomes may vary, and the treatment requires careful monitoring, say haematologists.

Dr Nita Radhakrishnan, Additional Professor and Head, Department of Paediatric Haematology-Oncology, Post Graduate Institute of Child Health (PGICH), Noida, said, "The availability of new therapies such as fitusiran is an exciting development for people living with haemophilia A and B, with or without inhibitors, offering a broad treatment approach across both conditions.”

“Subcutaneous injection once every two months can reduce the burden of frequent intravenous infusions while improving bleed prevention and potentially reducing joint damage and long-term disability.”

Dr Mukul Aggarwal, Additional Professor of Clinical Haematology, Department of Haematology, AIIMS New Delhi, said haemophilia treatment had evolved from blood and plasma transfusions to plasma-derived and recombinant clotting-factor concentrates.

“All these are given intravenously, 2-3 times a day for bleeding control and 2-3 times a week to prevent bleeding as prophylaxis. Still, breakthrough bleeding can occur 1-5 times a year.

"Talking about the advancement in the sector, he pointed out that the last decade has seen the evolution of non-factor therapies like Emicizumab, Concizumab, and now Fitusiran, which have been approved by regulatory authorities for patient use. All these are given subcutaneously, a far more convenient route of administration, from once a day to once a month, with excellent bleed control to 1-3 episodes per year.

“Fitusiran, which was approved by the FDA in 2025 and now by India’s top drug regulator, is an antithrombin-directed small interfering ribonucleic acid (RNA) indicated for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and paediatric patients aged 12 years and older with haemophilia.

“Antithrombin is a naturally occurring blood thinner, and by reducing its blood levels, Fitusiran decreases bleeding in haemophilia patients. A major advantage is its utility in both haemophilia A and B, with or without factor VIII or IX antibodies to factor inhibitors.

“It is to be given every 2 months, i.e., your phone and company will remind you only 6 times a year for a subcutaneous jab. The manufacturer mandates monitoring it once with a blood test – antithrombin level – to adjust the dose, for which the patient does not pay.”

Dr Aggarwal, however, cautioned that, “As this is a new drug for the sector in India, monitoring is required for bleeding events and its side effects of thrombosis and gallbladder and liver toxicity.

“If required, patients can undergo surgical procedures as well, at experienced centres under predefined protocols. It is going to be an excellent tool in our armamentarium for haemophilia management.”

Dr Mayank Nilay, Associate Professor and Head of Medical Genetics at PGICH, Noida, explained the treatment’s mechanism. “It is an RNAi-based treatment that modifies the expression of a target gene, SERPINC1, to reduce antithrombin production. It does not correct the inherited F8 or F9 defect.

“RNA interference (RNAi) therapy is a treatment that switches down the production of a specific protein by targeting its messenger RNA (mRNA),” he added.

The drug’s mechanism also makes safety monitoring crucial. Reducing antithrombin can increase the risk of excessive clotting and potentially serious thrombotic events. The US prescribing information calls for liver-function tests at baseline and monthly for at least six months after starting treatment and after dose increases, followed by periodic monitoring. The precise monitoring requirements under the Indian authorisation should be checked against the approved prescribing information.

Patients should not start, stop or change treatment without a specialist’s advice. Breakthrough bleeding or suspected complications require prompt medical assessment, said the health experts.

India has an estimated 1.5 lakh people living with haemophilia, according to the Indian Council of Medical Research (ICMR). However, only about 27,000 cases were diagnosed and registered with the Haemophilia Federation of India, highlighting a substantial gap in diagnosis and registration. Limited access to specialised testing and awareness are among the barriers to identifying patients.

For many families, the challenge extends beyond diagnosis to sustaining treatment and preventing long-term complications. The new therapy could offer another preventive option, but its impact will depend on its price, availability and whether healthcare facilities can provide the necessary monitoring, said the stakeholders.

Vaibhav Nehra, Secretary, Haemophilia Society Delhi, called it a new-age non-factor therapy. “It will be helpful in enhancing preventive care for persons with haemophilia. I hope to see states adopt this along with other non-factor therapies quickly so that the level of care in India enhances.”

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 haemostasis and the power of lowering it to rebalance the coagulation system… The dosing of Qfitlia is guided by AT levels – a measurable, reliable biomarker that allows physicians to personalise treatment and optimise the risk-benefit profile for each person living with haemophilia."

Noting that a significant proportion of patients currently receiving treatment in India were still on-demand therapy, Parwas Mahajan, Business Head, Rare Diseases, Sanofi India, said, “The approval expands prophylactic treatment options for people living with haemophilia in India… and today's approval of Qfitlia is a significant step toward making that a reality.”