By Fineline Cube 
Pfizer Inc. (NYSE: PFE) announced that Hympavzi (marstacimab), the world’s first non‑factor hemophilia therapy requiring only a once‑weekly fixed‑dose subcutaneous injection, has received marketing approval from China’s National Medical Products Administration (NMPA) for routine prophylaxis in patients aged ≥12 years and weighing ≥35 kg with severe Hemophilia A or B without inhibitors.
Regulatory Milestone
| Item | Detail |
|---|---|
| Product | Hympavzi (marstacimab) |
| Company | Pfizer Inc. (NYSE: PFE) |
| Agency | NMPA (China) |
| Approval Type | Marketing authorization (first‑in‑class) |
| Indications | Severe Hemophilia A (FVIII < 1%) or Hemophilia B (FIX < 1%) without inhibitors |
| Age/Weight | ≥12 years, ≥35 kg |
| Key Innovation | Once‑weekly subcutaneous injection vs. IV factor replacement |
| Next Steps | Launch expected Q1 2026; reimbursement negotiations underway |
Drug Profile
- Mechanism: Non‑factor therapy targeting Tissue Factor Pathway Inhibitor (TFPI) to rebalance coagulation, bypassing need for factor VIII or IX replacement
- Administration: Fixed‑dose subcutaneous injection once weekly vs. traditional IV factor infusion (multiple times weekly)
- Dual Indication: Single product treats both Hemophilia A and B, simplifying inventory and prescribing
- Clinical Burden Reduction: Subcutaneous route eliminates need for venous access and reduces infusion time from hours to minutes
Clinical Evidence Summary
- Basis: Phase III BASIS study demonstrated 91% reduction in treated bleeds vs. on‑demand factor therapy; non‑inferior to prophylactic factor in Hemophilia A and B patients without inhibitors
- Safety: Consistent with mechanism; no unexpected thrombotic events; injection‑site reactions in 15% of patients (mild‑moderate)
- Patient Preference: 85% of trial participants preferred subcutaneous weekly dosing over IV factor regimens
Market Impact & Outlook
| Metric | Value |
|---|---|
| China Hemophilia Patients (Severe A/B) | ~30,000 patients (A: 22,000; B: 8,000) |
| Current Standard | Factor replacement (IV) 2‑3× weekly; costs ¥200,000‑400,000/year |
| Hympavzi Pricing | Estimated ¥250,000‑300,000/year (aligned with factor prophylaxis) |
| Market Penetration Target | 20‑30% of eligible patients by 2028 |
| Peak Sales Forecast (China) | ¥1.5‑2.0 billion (US$200‑270 million) by 2029 |
| Global Context | Approved in US (FDA, Oct 2024) and EU (Feb 2025); China approval completes major market access |
- Reimbursement Path: Expected to be included in China National Reimbursement Drug List (NRDL) by 2027 based on novel mechanism and patient convenience; Pfizer is prepared for price negotiation.
- Competitive Moat: First and only weekly subcutaneous non‑factor therapy; no biosimilar pathway; patent protection through 2038 in China.
- Strategic Value: Strengthens Pfizer’s rare disease portfolio in China, complementing BeneFIX (FIX) and Xyntha (FVIII) while addressing the 15‑20% of patients who develop inhibitors.
Forward‑Looking Statements
This brief contains forward‑looking statements regarding Hympavzi’s commercial launch, reimbursement negotiations, and market penetration in China. Actual results may differ materially due to risks including pricing negotiations, competitive responses, and patient adoption rates.-Fineline Info & Tech