By Fineline Cube 
HutchMed (China) Ltd (NASDAQ: HCM, HKG: 0013) announced that China’s National Medical Products Administration (NMPA) has accepted the New Drug Application (NDA) for fanregratinib (HMPL‑453) and granted Priority Review for the treatment of adult patients with advanced, metastatic, or unresectable intrahepatic cholangiocarcinoma (iCCA) harboring FGFR2 fusion or rearrangement after prior systemic therapy.
Regulatory Milestone
| Item | Detail |
|---|---|
| Product | Fanregratinib (HMPL-453) |
| Company | HutchMed (China) Ltd (NASDAQ: HCM, HKG: 0013) |
| Mechanism | Highly selective FGFR 1, 2, and 3 inhibitor |
| Indication | FGFR2 fusion/rearrangement-positive iCCA (post-systemic therapy) |
| Regulatory Status | NDA accepted with Priority Review (China) |
| Study Support | Single-arm, multi-center, open-label Phase II registration study |
| Primary Endpoint | Objective Response Rate (ORR) – met |
Clinical Evidence – Phase II Study
| Endpoint | Result | Clinical Significance |
|---|---|---|
| Objective Response Rate (ORR) | Primary endpoint met | Demonstrates anti-tumor activity |
| Progression-Free Survival (PFS) | Secondary endpoint supportive | Duration of disease control |
| Disease Control Rate (DCR) | Secondary endpoint supportive | Overall clinical benefit |
| Duration of Response (DoR) | Secondary endpoint supportive | Sustainability of responses |
| Overall Survival (OS) | Secondary endpoint supportive | Long-term survival benefit |
Study Design: Single-arm, multi-center, open-label Phase II registration study conducted in China.
Market Context & Opportunity
Intrahepatic Cholangiocarcinoma (iCCA) Landscape:
- Incidence: Accounts for 8.2% to 15.0% of primary liver cancers, making it the second most common after hepatocellular carcinoma (HCC)
- Global Trend: Incidence steadily increasing worldwide
- Prognosis: 5-year overall survival rate ~9%, highlighting urgent need for effective therapies
- FGFR2 Prevalence: 10% to 15% of iCCA patients harbor FGFR2 fusions/rearrangements, representing a targetable molecular subset
| Parameter | China | Global |
|---|---|---|
| Annual iCCA incidence | ~45,000 cases | ~200,000 cases |
| FGFR2+ addressable population | 4,500-6,750 patients | 20,000-30,000 patients |
| Current SOC | Chemotherapy, limited targeted options | Pemigatinib (US/EU), Infigratinib (US) |
| Market Gap | No approved FGFR inhibitors in China | – |
Competitive Landscape
| Drug | Company | Target | Status in China | Differentiation |
|---|---|---|---|---|
| Fanregratinib | HutchMed | FGFR 1/2/3 | NDA accepted (Priority Review) | First potential FGFR inhibitor for iCCA in China |
| Pemigatinib | Incyte/信达 | FGFR 1/2/3 | Phase III ongoing | Approved in US/EU; not yet in China |
| Infigratinib | BridgeBio/QED | FGFR 1/2/3 | Not filed | Approved in US; limited data in Asian patients |
| Futibatinib | Taiho | FGFR 1-4 | Not filed | FGFR4 activity may increase toxicity |
Strategic Position: Fanregratinib could be first-to-market in China, capturing an estimated 60-70% market share in FGFR2+ iCCA.
Financial Outlook
| Parameter | 2027E | 2029E | 2031E |
|---|---|---|---|
| China iCCA patients (FGFR2+) | 5,500 | 6,200 | 7,000 |
| Fanregratinib penetration | 40% | 55% | 65% |
| Annual therapy cost (¥) | ¥280,000 | ¥240,000 (post-NRDL) | ¥200,000 |
| Peak China sales | ¥616 million | ¥818 million | ¥910 million (US$127M) |
| Global expansion potential | – | US/EU filing | Additional ¥1.5B upside |
Forward‑Looking Statements
This brief contains forward‑looking statements regarding fanregratinib’s regulatory timeline, market potential, and clinical development plans. Actual results may differ materially due to regulatory review outcomes, competitive dynamics, and market access challenges.-Fineline Info & Tech