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CDSCO Approves Qfitlia for Haemophilia Bleed Prevention
What’s the News?
The Central Drugs Standard Control Organisation (CDSCO) has granted marketing authorisation to Qfitlia (Fitusiran), developed by French pharmaceutical company Sanofi, for preventing bleeding episodes in patients with haemophilia A and B.
It is India's first approved antithrombin-lowering therapy for routine bleeding prevention in adults and adolescents aged 12 years and above, including patients with or without clotting-factor inhibitors.
About Qfitlia (Fitusiran)
- Developer: Sanofi Healthcare.
- Active Ingredient: Fitusiran.
- Technology: Small interfering RNA (siRNA) therapy.
- Mechanism: Reduces antithrombin, a natural anticoagulant protein, to improve blood clotting without replacing deficient clotting factors.
- Administration: Subcutaneous injection, requiring as few as 6 injections annually, depending on individual dosing.
- Clinical Evidence: Approval was based on the ATLAS Phase III clinical development programme, which demonstrated a reduction in bleeding episodes.
- Significance: Provides preventive treatment for both haemophilia A and B, including patients who develop inhibitors against conventional clotting-factor replacement therapies.
What is Haemophilia?
- Haemophilia is a rare, inherited, lifelong bleeding disorder caused by deficiency or dysfunction of specific blood-clotting factors.
- It primarily affects males due to its X-linked recessive inheritance pattern.
- Major Types:
- Haemophilia A: Deficiency of clotting factor VIII.
- Haemophilia B: Deficiency of clotting factor IX.
- Symptoms: Prolonged bleeding, spontaneous internal bleeding and joint damage.
- India's Burden: Approximately 28,900 registered haemophilia patients.
- Treatment Approaches:
- On-demand therapy: Treats bleeding after it occurs.
- Prophylaxis: Preventive treatment to reduce bleeding episodes.
Additional Info
- CDSCO (Central Drugs Standard Control Organisation):
- India's national regulatory authority for drugs and medical devices.
- Functions under the Directorate General of Health Services (DGHS), Ministry of Health and Family Welfare.
- Headed by the Drugs Controller General of India (DCGI).
- Responsible for drug approvals, clinical trial oversight and regulation of specified categories of drugs.
- World Haemophilia Day:
- Observed annually on 17 April.
- Organised globally by the World Federation of Hemophilia (WFH).