By Fineline Cube AstraZeneca PLC (AZ, NASDAQ: AZN) and Ionis Pharmaceuticals (NASDAQ: IONS) announced that Wainua (eplontersen) has received approval from China’s National Medical Products Administration (NMPA) for the treatment of adult patients with hereditary transthyretin-mediated amyloidosis with polyneuropathy (ATTRv-PN), marking the first and only gene-silencing agent cleared for this indication in China.
Regulatory Milestone
| Item | Detail |
|---|---|
| Product | Wainua (eplontersen) |
| Mechanism | RNA-targeted gene-silencing agent (antisense oligonucleotide) |
| Indication | Hereditary transthyretin-mediated amyloidosis in adults (ATTRv-PN) |
| Regulator | NMPA (China) |
| Approval Date | 25 Dec 2025 |
| Significance | First approved gene-silencing therapy for ATTRv-PN in China; self-administered monthly via auto-injector |
Disease Profile & Unmet Need
- Prevalence: ~30,000-50,000 affected individuals in China; diagnosis rate <5%
- Clinical Course: Motor dysfunction within 5 years post-diagnosis; median survival <10 years without disease-modifying treatment
- Economic Burden: Annual direct healthcare costs ¥200,000-300,000 per patient; indirect disability costs exceed ¥400,000
- Current Landscape: No approved disease-modifying therapies; limited to symptomatic management and liver transplant in select cases
Clinical Evidence – NEURO‑TTRansform Phase 3
| Endpoint | Eplontersen (66 weeks) | External Placebo | Clinical Significance |
|---|---|---|---|
| Co‑Primary: Serum TTR Concentration | >80% sustained reduction | Minimal change | Direct target engagement |
| Co‑Primary: Neuropathy (mNIS+7) | Stable vs. baseline | Progressive deterioration | Halts disease progression |
| Key Secondary: QoL (Norfolk QoL‑DN) | Sustained improvement | Declining scores | Patient‑reported benefit |
| Safety Profile | Well‑tolerated; 4% discontinuation due to AEs | – | Favorable long‑term safety |
Study Design: Randomized, controlled Phase 3 trial; 66‑week observation period vs. external placebo comparator.
Drug Profile & Strategic Position
- Mechanism: Targets transthyretin (TTR) mRNA; upstream inhibition of pathogenic protein production
- Administration: Self‑administered monthly subcutaneous injection via auto‑injector; enables home treatment
- Global Rollout: Approved US (FDA Dec 2023), EU (Mar 2024); China approval completes major market entry
- IP & Partnership: Licensed from Ionis; AstraZeneca holds full commercial rights in Asia; co‑promotion strategy leverages AZ’s rare disease infrastructure
Market Opportunity & Financial Outlook
| Parameter | 2026E | 2028E |
|---|---|---|
| Diagnosed ATTRv‑PN patients in China | 2,500 | 4,500 |
| Eplontersen‑eligible population | 2,000 | 3,600 |
| Peak market penetration | 35% | 55% |
| Annual treatment cost (¥) | 380,000 | 320,000 (post‑NRDL) |
| Estimated China revenue | ¥266 million | ¥633 million (US$37‑88 million) |
- Launch Timeline: Q1 2026 commercial rollout via specialty pharmacy channels
- Reimbursement Strategy: Targeting 2026 NRDL negotiation; orphan drug designation supports premium pricing
- Patient Support: “Wainua Care” program launching Q1 2026 with co‑pay assistance and genetic counseling services
- Diagnostic Push: Partnering with BGI Genomics to expand TTR gene testing capacity and accelerate diagnosis
Forward‑Looking Statements
This brief contains forward‑looking projections regarding eplontersen’s market penetration, revenue potential, and reimbursement timelines in China. Actual results may differ materially due to competitive responses, diagnostic infrastructure limitations, and NRDL negotiation outcomes.-Fineline Info & Tech