Sanofi Receives Indian Approval for Qfitlia, a New RNA-Based Treatment for Haemophilia A and B

India has approved Qfitlia (fitusiran), an innovative RNA-based medicine developed by global biopharmaceutical company Sanofi, for the prevention of bleeding episodes in patients with haemophilia A or haemophilia B. The approval by the Central Drugs Standard Control Organisation (CDSCO), announced on 21 October 2026, introduces a new preventive treatment option for patients aged 12 years and above, including those who have developed inhibitors against conventional clotting factor therapies.

Qfitlia represents an important development in haemophilia treatment because it works differently from traditional medicines that replace missing blood-clotting factors. Instead, the therapy uses small interfering RNA (siRNA) technology to reduce the production of antithrombin, a naturally occurring protein that regulates blood coagulation. By carefully lowering antithrombin activity, the medicine helps restore a more effective balance in the clotting process.

The treatment is administered through subcutaneous injections, with an individualised dosing schedule guided by regular monitoring of antithrombin activity. According to Sanofi, eligible patients may receive treatment as infrequently as once every two months, potentially reducing the number of preventive injections to six annually.

The Indian approval follows encouraging results from the international Phase III ATLAS clinical development programme and regulatory authorisation in other major pharmaceutical markets, including the United States. It expands the availability of advanced non-factor therapies for people living with inherited bleeding disorders in India.

CDSCO Approves Qfitlia for Patients Aged 12 Years and Above

The Central Drugs Standard Control Organisation has authorised Qfitlia for routine prophylaxis to prevent or reduce bleeding episodes in patients aged 12 years and older with haemophilia A or B.

An important feature of the approval is that the treatment covers patients both with and without inhibitors against clotting factors VIII or IX. This distinction is particularly significant because the development of inhibitors can complicate conventional haemophilia treatment.

Sanofi Healthcare India had presented its application for permission to import and market fitusiran to the Subject Expert Committee for Haematology at a meeting held on 13 January 2026.

The application covered fitusiran solution for subcutaneous injection in two presentations: a 50 mg/0.5 mL prefilled pen and a 20 mg/0.2 mL vial.

During its review, the committee examined international Phase III clinical trial data and information concerning the participation of Indian patients in the global development programme. It also considered the medicine’s existing regulatory approvals in the United States and the United Arab Emirates.

The committee recommended permission for import and marketing, subject to safety-monitoring and post-marketing requirements. It directed the company to conduct a post-marketing surveillance study and submit an appropriate protocol to CDSCO within three months of receiving marketing authorisation.

The committee also requested updated prescribing information containing relevant adverse reactions and safety warnings consistent with internationally approved product information.

The recommendation formed an important regulatory step preceding the company’s October announcement of approval in India.

Understanding Haemophilia A and Haemophilia B

Haemophilia is an inherited bleeding disorder in which the blood does not clot normally because of deficiencies or abnormalities involving specific clotting proteins.

Haemophilia A results from insufficient or defective clotting factor VIII, while haemophilia B is associated with a deficiency of clotting factor IX. Both proteins play essential roles in the series of biochemical reactions that allow blood to form stable clots after an injury.

People with haemophilia may experience prolonged bleeding following injuries, medical procedures or surgery. Those with severe disease can also develop spontaneous bleeding episodes without an obvious external injury.

Repeated bleeding into joints is particularly concerning because it can cause pain, swelling, reduced mobility and progressive joint damage. Bleeding into muscles and internal organs can lead to additional complications, while intracranial bleeding may become life-threatening.

The disorders primarily affect males because the genes responsible for haemophilia A and B are located on the X chromosome. However, females who carry haemophilia-related genetic variants can also experience bleeding symptoms, sometimes associated with reduced clotting factor activity.

The severity of haemophilia varies according to the level of functional clotting factor present in the blood. Patients with severe disease often require regular preventive treatment to reduce spontaneous bleeding and protect long-term joint health.

Modern haemophilia care therefore places considerable emphasis on prophylaxis, which involves administering treatment before bleeding occurs rather than responding only after an episode begins.

How Qfitlia Uses RNA Interference to Restore Clotting Balance

Qfitlia introduces a different approach to managing haemophilia by targeting the body’s natural anticoagulation system.

The medicine contains fitusiran, a small interfering RNA molecule designed to reduce the production of antithrombin in the liver.

Antithrombin is a naturally occurring protein that limits blood clotting by inhibiting several clotting enzymes, particularly thrombin and factor Xa. It plays an important role in preventing excessive clot formation under normal physiological conditions.

In patients with haemophilia, insufficient factor VIII or factor IX activity disrupts the generation of thrombin, an enzyme required to convert fibrinogen into fibrin and produce a stable blood clot.

Fitusiran works by reducing the synthesis of antithrombin, thereby allowing greater thrombin generation despite the underlying clotting factor deficiency.

The medicine achieves this through RNA interference, a biological mechanism in which small RNA molecules reduce the expression of selected genes.

After entering liver cells, fitusiran targets messenger RNA involved in antithrombin production. This decreases the amount of antithrombin protein released into the bloodstream.

By carefully regulating this effect, treatment can improve the balance between clot-promoting and clot-inhibiting processes.

This approach is known as a rebalancing therapy because it seeks to restore more effective clot formation without directly replacing the missing clotting factor.

Unlike gene therapy, fitusiran does not permanently alter a patient’s DNA or correct the inherited genetic defect responsible for haemophilia. Its therapeutic effect requires continued treatment and monitoring.

A Non-Factor Treatment for Both Major Forms of Haemophilia

Conventional haemophilia management has historically relied on replacing the deficient clotting factor.

Patients with haemophilia A may receive factor VIII replacement therapy, while those with haemophilia B may receive factor IX concentrates. The required frequency depends on the preparation used, the severity of the condition and individual clinical circumstances.

Although these treatments can be effective, some patients develop inhibitory antibodies against the administered clotting factors.

These antibodies can reduce or neutralise the effectiveness of replacement therapy, making bleeding episodes more difficult to prevent and manage.

Fitusiran offers an alternative mechanism because it does not depend on supplying factor VIII or factor IX. Its antithrombin-lowering action can support thrombin generation in patients with either form of haemophilia.

This characteristic allows the medicine to be used in eligible patients regardless of whether they have developed inhibitors.

The treatment’s applicability to both haemophilia A and haemophilia B distinguishes it from some other non-factor prophylactic therapies that are specific to haemophilia A.

However, fitusiran does not eliminate the underlying disease, and patients may still require additional treatment for breakthrough bleeding, injuries or surgical procedures.

Such treatment must follow specialised clinical guidance because the altered coagulation balance affects how other haemostatic medicines should be administered.

Phase III ATLAS Programme Demonstrates Reduced Bleeding Episodes

The clinical evidence supporting Qfitlia includes the international Phase III ATLAS programme, which evaluated fitusiran in patients with haemophilia A and B.

The development programme included studies involving patients with inhibitors, patients without inhibitors and participants receiving different haemophilia treatment regimens.

Two randomised trials compared fitusiran with conventional on-demand treatment. Patients subsequently entered a long-term extension study, ATLAS-OLE, in which investigators evaluated an individualised dosing approach based on antithrombin activity.

The United States Food and Drug Administration approved Qfitlia on 28 March 2025 after reviewing the available clinical evidence.

According to the FDA, patients with inhibitors who received the antithrombin-guided dosing regimen experienced a 73% reduction in the estimated annualised bleeding rate compared with patients receiving on-demand treatment using bypassing agents.

Among patients without inhibitors, the corresponding reduction was 71% compared with on-demand clotting factor treatment.

These results provide evidence that appropriately monitored antithrombin-lowering therapy can substantially reduce bleeding episodes in eligible patients.

The findings describe relative reductions in estimated bleeding rates between treatment groups. They do not mean that every patient became free from bleeding or that the treatment permanently corrected haemophilia.

The FDA’s assessment also identified important safety considerations that influenced the development of the individualised dosing strategy.

Personalised Dosing Through Antithrombin Monitoring

One of Qfitlia’s important features is its individualised dosing approach, which uses antithrombin activity as a biomarker to guide treatment.

Rather than administering the same fixed dose to every patient throughout treatment, clinicians monitor antithrombin levels and adjust the medicine’s dosage or administration interval to maintain an appropriate coagulation balance.

The USFDA-approved regimen begins with a 50 mg subcutaneous injection once every two months. Subsequent adjustments depend on the patient’s antithrombin activity and clinical requirements.

Under the American prescribing information, treatment aims to maintain antithrombin activity within a target range of 15% to 35%. This approach seeks to reduce bleeding while limiting the risk of excessive clot formation.

The use of a measurable biological marker provides a means of tailoring treatment according to individual responses. Patients whose antithrombin activity changes differently can receive adjusted treatment schedules rather than continuing with a fixed regimen.

The Indian prescribing information and the treating specialist’s instructions govern the appropriate dose and monitoring schedule for patients receiving Qfitlia in India.

Regular laboratory monitoring remains essential because excessive suppression of antithrombin can increase the risk of abnormal blood clotting.

Potential to Reduce the Frequency of Preventive Injections

For people living with severe haemophilia, the frequency of treatment can influence everyday life.

Depending on the therapy, conventional prophylaxis may require repeated intravenous infusions or subcutaneous injections at varying intervals. Treatment schedules can affect school attendance, employment, travel and other routine activities.

Qfitlia’s subcutaneous administration offers an alternative to treatments requiring frequent intravenous access.

According to Sanofi, the medicine’s individualised dosing approach can allow some patients to receive preventive treatment as infrequently as once every two months.

This represents the possibility of approximately six injections annually for patients whose treatment remains suitable for that dosing interval.

The reduced injection frequency could lessen the practical burden associated with long-term preventive therapy. However, patients must continue attending medical appointments and undergoing laboratory investigations to maintain treatment safety.

The medicine’s suitability also depends on individual health conditions, bleeding history and the ability to monitor antithrombin activity appropriately.

The development therefore introduces greater flexibility into haemophilia treatment while retaining the need for specialist supervision.

Managing the Risks Associated with Antithrombin-Lowering Therapy

Although Qfitlia has demonstrated substantial reductions in bleeding episodes, its mechanism of action introduces important safety considerations.

Antithrombin normally protects the body against excessive coagulation. Reducing its activity can improve clot formation in patients with haemophilia, but excessive reduction may increase the risk of dangerous blood clots.

The United States prescribing information for Qfitlia carries boxed warnings concerning serious thrombotic events and acute or recurrent gallbladder disease.

Thrombotic complications can occur when abnormal clots develop within blood vessels and obstruct circulation. Such events may become serious or life-threatening.

Gallbladder complications have also occurred in patients receiving fitusiran. Some cases have required surgical removal of the gallbladder.

Liver-related adverse reactions represent another important concern. Patients receiving the medicine require liver-function assessment before treatment and periodic monitoring during therapy.

The FDA recommends liver blood tests at baseline and monthly for at least six months after treatment initiation or a dose increase, followed by further monitoring as clinically indicated.

The most frequently reported adverse reactions include viral infections, nasopharyngitis and bacterial infections. Other reported reactions include liver enzyme abnormalities, joint pain, injection-site reactions, headache and cough.

These risks highlight the importance of administering Qfitlia under the supervision of physicians experienced in treating haemophilia.

Appropriate patient selection, laboratory monitoring and careful management of breakthrough bleeding are essential components of safe treatment.

India’s Haemophilia Treatment Landscape

India has a substantial population of people living with inherited bleeding disorders, including haemophilia A and B.

According to figures cited in the October 2026 approval announcement, approximately 28,900 patients with haemophilia have been registered in India.

The registered population does not necessarily represent every person living with the condition. Diagnosis, access to specialised testing and the availability of treatment services vary across regions.

People with haemophilia often require long-term care involving haematologists, diagnostic laboratories, physiotherapy services and specialised treatment centres.

Preventing recurrent bleeding is particularly important because repeated joint haemorrhages can lead to chronic pain, reduced mobility and permanent disability.

The introduction of newer preventive medicines has expanded treatment options beyond traditional clotting factor replacement.

Nevertheless, the availability of advanced therapies depends on regulatory approval, distribution arrangements, medical infrastructure, affordability and the ability of healthcare facilities to provide appropriate monitoring.

The approval of Qfitlia adds another option to India’s haemophilia treatment landscape, particularly for patients whose clinical circumstances make non-factor prophylaxis suitable.

International Regulatory Recognition of Qfitlia

Qfitlia received its first approval from the United States Food and Drug Administration on 28 March 2025.

The American regulator authorised the medicine for routine prophylaxis in adults and children aged 12 years and older with haemophilia A or B, regardless of inhibitor status.

The approval followed an assessment of clinical evidence supporting antithrombin-guided dosing and the treatment’s ability to reduce bleeding episodes.

Sanofi subsequently secured approvals in additional international markets.

In December 2025, China’s National Medical Products Administration approved Qfitlia as part of the company’s expanding rare-disease treatment portfolio.

The Indian regulatory process also considered the medicine’s international clinical evidence and existing approvals. During the January 2026 review, CDSCO’s Haematology Subject Expert Committee examined the participation of Indian patients in the international Phase III programme.

The committee recognised the unmet medical need associated with haemophilia treatment in India and recommended approval subject to post-marketing safety requirements.

The subsequent Indian approval expands the medicine’s availability within another major healthcare market.

Advanced RNA Technology Expands Its Role in Medicine

The development of fitusiran illustrates the growing therapeutic applications of RNA interference technology.

RNA interference is a natural cellular mechanism through which selected RNA molecules regulate the expression of genes. Scientists have adapted this process to develop medicines that reduce the production of proteins involved in particular diseases.

Small interfering RNA medicines contain specially designed molecules that bind to specific messenger RNA sequences. This interaction can lead to the degradation of the targeted RNA, reducing the production of the corresponding protein.

In the case of fitusiran, the target is antithrombin, a protein produced primarily in the liver.

The medicine’s ability to reduce antithrombin production demonstrates how RNA-based technologies can influence biological processes without directly replacing a missing protein.

Other RNA interference medicines have received regulatory approval for conditions involving abnormal protein production or inherited metabolic disorders.

These developments represent an expanding field of precision therapeutics in which treatments target defined molecular pathways.

For haemophilia, this approach is especially significant because it offers a way to improve coagulation despite the continued presence of the underlying genetic factor deficiency.

Qfitlia therefore represents an example of advanced RNA technology being translated into an approved preventive treatment for a chronic inherited disorder.

Strengthening Rare-Disease Treatment in India

The introduction of new treatments for rare diseases is becoming an important component of India’s evolving healthcare landscape.

Rare and inherited disorders frequently require specialised diagnosis, long-term medical supervision and therapies developed for relatively small patient populations.

Haemophilia is an example of a condition in which advances in biotechnology have progressively changed treatment approaches.

Improved clotting factor concentrates, extended-half-life products and non-factor therapies have created additional options for preventing bleeding and reducing long-term complications.

Qfitlia expands this range by introducing antithrombin-lowering therapy for eligible patients with either haemophilia A or haemophilia B.

The January 2026 CDSCO committee recommendation also emphasised the importance of post-marketing surveillance, reflecting the need to monitor the medicine’s safety as it becomes available to a wider patient population.

Continued physician education, appropriate laboratory facilities and patient awareness will be important for supporting its clinical use.

The development highlights the contribution of advanced biopharmaceutical research to improving treatment choices for patients with inherited medical conditions.

A New Chapter in Preventive Haemophilia Care

The CDSCO approval of Sanofi’s Qfitlia introduces an additional preventive treatment option for patients aged 12 years and above with haemophilia A or B in India.

Its RNA interference mechanism offers a different approach from conventional clotting factor replacement. By reducing antithrombin production, the medicine helps restore the balance needed for more effective blood coagulation.

Clinical evidence from the ATLAS programme demonstrates that antithrombin-guided treatment can substantially reduce bleeding episodes compared with on-demand therapies in the studied populations.

The possibility of less frequent subcutaneous injections may also reduce the practical burden of long-term prophylaxis for eligible patients.

At the same time, careful monitoring remains essential because the treatment can cause serious adverse reactions, including blood clots, gallbladder complications and liver injury.

The Indian approval reflects the continued progress of RNA-based medicines and expands the range of specialised therapies available for inherited bleeding disorders.

As healthcare providers strengthen awareness, diagnosis and access to preventive treatment, innovations such as Qfitlia can contribute to improving long-term haemophilia management and reducing the complications associated with recurrent bleeding.

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References

1. Central Drugs Standard Control Organisation – Haematology Subject Expert Committee Recommendations, 13 January 2026

Government of India.

Official regulatory record concerning Sanofi Healthcare India’s application for permission to import and market fitusiran. The committee recommended approval subject to post-marketing surveillance and updated prescribing information.

https://cdsco.gov.in/opencms/resources/UploadCDSCOWeb/2018/UploadCommitteeFiles/Recommendations%20Haematology%2013.01.2026.pdf

2. United States Food and Drug Administration – FDA Approves Novel Treatment for Haemophilia A or B

28 March 2025.

Official announcement covering the approval of Qfitlia, its antithrombin-lowering mechanism, clinical effectiveness, individualised dosing and safety considerations.

https://www.fda.gov/news-events/press-announcements/fda-approves-novel-treatment-hemophilia-or-b-or-without-factor-inhibitors

3. United States Food and Drug Administration – Qfitlia Drug Trials Snapshot

Official assessment of clinical trial participants, the ATLAS-OLE extension study, efficacy outcomes and reported adverse reactions.

https://www.fda.gov/drugs/drug-trials-snapshots/drug-trials-snapshots-qfitlia

4. United States Food and Drug Administration – Qfitlia Prescribing Information

Official regulatory document covering indications, administration, antithrombin monitoring, adverse reactions and boxed safety warnings.

https://www.accessdata.fda.gov/drugsatfda_docs/label/2025/219019s000lbl.pdf

5. Sanofi – Qfitlia and Cablivi Approved in China, Expanding Care for Rare Diseases

11 December 2025.

Official Sanofi announcement confirming Qfitlia’s international regulatory expansion and its potential for administration as infrequently as six injections annually.

https://www.sanofi.com/en/media-room/press-releases/2025/2025-12-11-06-00-00-3203611

6. Central Drugs Standard Control Organisation – Official Regulatory Portal

Government of India.

Official source for drug regulatory approvals, expert committee recommendations and pharmaceutical regulations.

https://cdsco.gov.in

7. Sanofi – Official Corporate Website

Information on Sanofi’s pharmaceutical research, rare-disease programmes and haemophilia treatments.

https://www.sanofi.com

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