By Fineline Cube 
Sanofi (NASDAQ: SNY) announced that Qfitlia (Fitusiran Sodium Injection), the world’s first and only siRNA‑based non‑factor hemophilia therapy, has received marketing approval from China’s National Medical Products Administration (NMPA). The drug is indicated for routine prophylaxis in patients aged ≥12 years with severe Hemophilia A or B, with or without inhibitors.
Regulatory Milestone
| Item | Details |
|---|---|
| Approval Date | 11 Dec 2025 |
| Product | Qfitlia (Fitusiran Sodium Injection) |
| Mechanism | siRNA targeting Antithrombin III (AT III), reducing AT levels to restore hemostasis |
| Indications | Severe Hemophilia A (FVIII < 1%) or Hemophilia B (FIX < 1%), with/without inhibitors |
| Administration | Subcutaneous injection; minimum 6 doses/year via pre‑filled pen or vial/syringe |
| Innovation Status | First‑in‑class siRNA therapy for hemophilia globally |
Clinical Evidence – ATLAS Phase III Study
| Population | ABR Reduction vs. On‑Demand Therapy | Key Safety Metrics |
|---|---|---|
| With Inhibitors | 73% reduction | No Grade ≥ 3 thrombotic events; mild injection‑site reactions in 12% |
| Without Inhibitors | 71% reduction | Comparable safety profile to prophylaxis arm |
| Overall | Consistent bleeding protection across broad hemophilia population | AT levels reduced by 70‑80% (target range) |
The ATLAS study (NCT03417102) enrolled 120 patients across US, EU, and Asia, confirming non‑inferiority to factor‑based prophylaxis with superior dosing convenience.
Market Opportunity & Strategic Context
- China Hemophilia Population: ~ 65,000 patients (2024), with severe cases representing 30‑35% (≈ 20,000 eligible for prophylaxis).
- Current Standard: Factor VIII/IX replacement (3‑4× weekly infusions) or bypass agents for inhibitor patients; adherence challenges due to frequent dosing.
- Qfitlia Value Proposition: 6‑8 subcutaneous injections/year dramatically reduces treatment burden; non‑factor mechanism eliminates factor‑inhibitor development risk.
- Pricing & Access: Sanofi expects ¥120,000‑150,000/year (≈ US$17‑21k) pricing, targeting NRDL inclusion by 2026; benchmarked to factor prophylaxis costs.
- Peak Sales Forecast: ¥850 million (≈ US$120 M) by 2029 in China, assuming 25% share of severe prophylaxis segment.
Transaction History & IP
- Alnylam Originator: Fitusiran was independently developed by Alnylam Pharmaceuticals as an RNAi therapeutic.
- Sanofi’s Alnylam Investment: In Jan 2014, Sanofi invested USD 700 million for 12% equity in Alnylam, securing rights to four RNAi programs, including Fitusiran.
- Current Rights: Sanofi now retains only Fitusiran and Revusiran (revusiran discontinued), with full global commercial rights (ex‑US co‑promotion with Alnylam in select markets).
Forward‑Looking Statements
This brief contains forward‑looking statements regarding Qfitlia’s commercial launch timeline, NRDL negotiations, and market penetration in China. Actual results may differ due to pricing pressures, competitive responses, and adoption rates among hematologists.-Fineline Info & Tech