By Fineline Cube 
Visen Pharmaceuticals (HKG: 2561), a joint venture between Ascendis Pharma (Denmark) and Vivo Capital, announced that China’s National Medical Products Administration (NMPA) has approved Skytrofa (lonapegsomatropin, TransCon hGH) for the treatment of growth failure due to growth hormone deficiency (GHD) in children and adolescents aged 3 years and older.
Regulatory Milestone
| Item | Detail |
|---|---|
| Company | Visen Pharmaceuticals (JV: Ascendis Pharma / Vivo Capital) |
| Drug | Skytrofa (lonapegsomatropin, TransCon hGH) |
| Approval | NMPA (China) – marketing authorization |
| Indication | Pediatric growth hormone deficiency (GHD) |
| Patient Population | Children and adolescents ≥3 years with growth failure |
| Technology | TransCon (Transient Conjugation) long‑acting formulation |
| Dosing | Weekly subcutaneous injection vs. conventional daily injections |
| Previous Approvals | Registered in Europe and the United States |
Clinical Profile & Unmet Need
- Disease Burden: Pediatric GHD is a serious rare disease where the pituitary gland fails to produce sufficient growth hormone, leading to significant growth failure
- Treatment Paradigm Shift: Lonapegsomatropin transforms the standard of care from daily to weekly injections, dramatically reducing treatment burden for pediatric patients and caregivers
- TransCon Technology: Proprietary transient conjugation technology enables sustained release of human growth hormone over 7 days while maintaining full biological activity
- Clinical Evidence: Pivotal Phase III trial in China demonstrated non‑inferiority to daily‑injected human growth hormone with comparable safety profile
Pipeline & Strategic Rights
- Exclusive Territory: Visen Pharma holds exclusive rights for development, manufacturing, and commercialization of Ascendis’s endocrinology pipeline in Greater China
- Portfolio Assets: Includes lonapegsomatropin (Skytrofa), palopegteriparatide, and navepegritide
- Market Position: First long‑acting weekly growth hormone approved in China, establishing Visen as leader in pediatric endocrinology
Market Impact & Commercial Outlook
- China Pediatric GHD Market: Estimated ¥8–10 billion (~US$1.1–1.4 billion) annually, with diagnosis rates increasing due to heightened awareness
- Competitive Advantage: Weekly dosing offers significant compliance benefit over daily injections from domestic competitors (GenSci, Anke Biotechnology)
- Pricing Strategy: Expected premium pricing at ¥150,000–200,000 per patient annually; NRDL negotiation anticipated for 2027 to expand access
- Revenue Forecast: Analysts project ¥1.5–2.0 billion (US$210–280 million) peak annual sales by 2029, capturing 15–20% market share
- Launch Timeline: Commercial availability expected Q2 2026 via specialty pharmacy and hospital channels
Forward‑Looking Statements
This brief contains forward‑looking statements regarding commercial expectations, market penetration, and revenue projections for Skytrofa in China. Actual results may differ due to pricing negotiations, competitive dynamics, and reimbursement decisions.-Fineline Info & Tech