By Fineline Cube 
Chiesi Group’s China unit announced that the National Medical Products Administration (NMPA) has granted marketing approval for Juxtapid (lomitapide) for the treatment of homozygous familial hypercholesterolemia (HoFH) in adult patients. The microsomal triglyceride transfer protein (MTP) inhibitor provides a new therapeutic option for patients who struggle to achieve lipid targets with conventional therapies.
Regulatory Milestone & Drug Profile
| Item | Detail |
|---|---|
| Company | Chiesi Group (China unit) |
| Drug | Juxtapid (lomitapide) |
| Mechanism | Microsomal triglyceride transfer protein (MTP) inhibitor |
| Approval | NMPA marketing authorization |
| Indication | HoFH in adults (combination with low-fat diet and other lipid-lowering therapies) |
| Key Efficacy | Reduces LDL-C, total cholesterol, apo B, and non-HDL-C |
| Innovation | Lipid-lowering effect independent of LDL receptor activity |
Mechanism of Action & Clinical Advantage
- MTP Inhibition: Lomitapide blocks MTP within hepatocytes and intestinal epithelial cells, preventing assembly of apo B‑containing VLDL and chylomicrons
- LDLR Independence: Works effectively even in HoFH patients with extremely low or absent residual LDL receptor function
- Additive Effect: Provides additional LDL‑C control on top of standard treatment through a different mechanism than statins or PCSK9 inhibitors
- Unmet Need: Addresses HoFH patients who have long struggled to achieve treatment targets, offering new potential for improving long-term prognosis
Market Impact & Commercial Outlook
- HoFH Prevalence: Estimated 1,000–2,000 patients in China; global prevalence of 1 in 250,000
- China Rare Disease Market: Valued at ¥30 billion : (~US$4.2 billion) in 2025, growing rapidly with improved diagnosis and orphan drug policies
- Pricing Advantage: HoFH drugs command premium pricing; Juxtapid could generate ¥300–500 million : (US$42–70 million) peak annual sales
- Competitive Landscape: Competes with evinacumab (Regeneron) and lomitapide (Aegerion); Chiesi’s local manufacturing and regulatory expertise offers market access advantage
- Strategic Value: Strengthens Chiesi’s rare disease portfolio in China; validates MTP inhibition approach for severe lipid disorders
- Next Steps: Commercial launch expected Q2 2026; patient access programs to be established for HoFH community
Forward‑Looking Statements
This brief contains forward‑looking statements regarding commercial launch timelines, market penetration, and revenue projections for Juxtapid. Actual results may differ due to competitive dynamics, pricing negotiations, and patient access challenges.-Fineline Info & Tech