By Fineline Cube 
Haihe Biopharma Co., Ltd. announced that risovalisib mesylate (CYH33), a highly potent and selective PI3Kα inhibitor, has been included in the Pilot Program to Encourage the Development of Pediatric Anti‑Tumor Drugs (SPARK Project) by China’s Center for Drug Evaluation (CDE). The designation covers PIK3CA‑Related Overgrowth Spectrum (PROS) and PIK3CA‑Related Vascular Malformations (PRVM) in pediatric patients requiring systemic therapy.
Regulatory Milestone
| Item | Detail |
|---|---|
| Product | Risovalisib mesylate (CYH33) |
| Company | Haihe Biopharma (full global IP rights) |
| Program | SPARK Project (Pediatric Anti‑Tumor Drug Development Pilot) |
| Indications | PROS and PRVM with severe clinical manifestations |
| Regulator | CDE, NMPA (China) |
| Designation Date | 25 Dec 2025 |
Drug Profile & Intellectual Property
- Mechanism: Highly selective PI3Kα inhibitor with novel chemical structure
- Intellectual Property: Full global IP rights held by Haihe Biopharma; no licensing obligations outside Japan
- Strategic Value: First‑in‑class oral small‑molecule targeting the PIK3CA pathway for rare pediatric overgrowth disorders
- Pediatric Focus: SPARK Project designation provides priority review, waived clinical trial fees, and dedicated CDE guidance
Clinical Development Status
| Geography | Indication | Status | Partner |
|---|---|---|---|
| Japan | PIK3CA‑mutant ovarian clear cell carcinoma (OCCC) | NDA submitted (Aug 2025) | Taiho Pharmaceutical (exclusive Japan rights, Oct 2025) |
| China | PROS/PRVM (pediatric) | SPARK Project inclusion (Dec 2025) | Haihe Biopharma (retains rights) |
| Global | Solid tumors (PIK3CA‑mutant) | Phase II ongoing | Haihe Biopharma |
Disease Market Context
PROS/PRVM Prevalence: Rare congenital disorders affecting 1‑2 per 100,000 births in China, translating to an estimated 2,000‑4,000 pediatric patients. Current standard of care is symptomatic management (surgery, compression therapy) with no approved systemic therapy.
| Parameter | 2026E | 2028E |
|---|---|---|
| Diagnosed pediatric PROS/PRVM patients (China) | 2,500 | 3,200 |
| Risovalisib‑eligible (severe, systemic) | 1,000 | 1,400 |
| Annual therapy cost (¥, pre‑reimbursement) | ¥250,000 | ¥200,000 |
| Peak penetration (2030E) | – | 60% |
| China revenue potential | – | ¥168 million (US$23 million) |
Orphan Drug Pathway: SPARK inclusion positions risovalisib for 2026‑2027 NRDL negotiation under rare disease provisions.
Competitive Landscape
| Drug | Company | Target | Indication | Stage |
|---|---|---|---|---|
| Risovalisib | Haihe Biopharma | PI3Kα | PROS/PRVM (pediatric) | SPARK Project |
| Alpelisib | Novartis | PI3Kα | Breast cancer | Marketed (adult only) |
| Copanlisib | Bayer | PI3Kα/δ | B‑cell lymphoma | Marketed (adult only) |
| Taselisib | Roche | PI3Kα | Solid tumors | Discontinued |
Advantage: Risovalisib is the only PI3Kα inhibitor in development specifically for pediatric PIK3CA‑related disorders, with global IP protection and regulatory fast‑track in China.
Forward‑Looking Statements
This brief contains forward‑looking statements regarding risovalisib’s regulatory pathway, market potential, and partnership dynamics with Taiho Pharmaceutical. Actual results may differ due to clinical trial outcomes, competitive responses, and NMPA pricing decisions.-Fineline Info & Tech