By Fineline Cube 
CANbridge Pharmaceuticals Inc. (HKG: 1228) announced that maralixibat has been approved by the Department of Health of Hong Kong, China for the treatment of cholestatic pruritus in patients aged 3 months and above with progressive familial intrahepatic cholestasis (PFIC).
Regulatory Milestone & Indication
| Attribute | Details |
|---|---|
| Product | maralixibat |
| Approval Authority | Department of Health, Hong Kong SAR |
| Indication | Cholestatic pruritus in PFIC patients |
| Age Eligibility | ≥3 months |
| Significance | First PFIC approval for CANbridge in Hong Kong; expands Greater China footprint |
Drug Origin & Mechanism of Action
| Element | Details |
|---|---|
| Original Discovery | Mirum Pharmaceuticals (US) |
| Drug Class | IBAT (Ileal Bile Acid Transporter) inhibitor |
| Mechanism | Blocks enterohepatic circulation of bile acids, reducing liver/serum bile acid levels and relieving pruritus |
| US Trade Name | Livmarli® |
| US FDA Approvals | ALGS (Sept 2021, age ≥1 year); PFIC (age ≥12 months) |
| China Approval | ALGS (June 2023, age ≥1 year) |
| Licensing | Exclusive Greater China rights secured by CANbridge (April 2021) |
Market Context & Competitive Landscape
- PFIC Prevalence: Rare pediatric cholestatic disease affecting ~1-2 per 100,000 births; significant unmet need in Asia.
- Hong Kong Market: Local approval enables market access and reimbursement pathways within the Greater China region.
- Competitive Position: No other IBAT inhibitors approved for PFIC in Hong Kong; Livmarli is established standard-of-care in US/EU.
- CANbridge Strategy: Builds on rare disease leadership; leverages existing ALGS infrastructure for PFIC launch.
Strategic Outlook & Financial Implications
- Revenue Model: Rare disease pricing expected at US$200,000-300,000 annual cost per patient; Hong Kong market potential estimated at US$5-10 million peak sales.
- Next Steps: CANbridge will pursue reimbursement listing in Hong Kong Hospital Authority; potential expansion to Macau and Taiwan.
- Pipeline Synergy: PFIC approval strengthens CANbridge’s pediatric liver disease franchise alongside ALGS; may enable lifecycle management for next-generation IBAT programs.
- Investor Value: Approval triggers milestone payment to Mirum; demonstrates CANbridge’s execution capability in licensed assets.
Forward‑Looking Statements
This brief contains forward‑looking statements regarding maralixibat’s commercial launch timeline, reimbursement outcomes, and market penetration in Hong Kong. Actual results may differ due to regulatory, pricing, and competitive risks.-Fineline Info & Tech