By Fineline Cube Chiesi Group announced that the first patients have initiated treatment with Raxone (idebenone 150 mg) at Ruijin Hainan Hospital, affiliated with Shanghai Jiao Tong University School of Medicine, less than one month after the drug received approval in the Boao Lecheng International Medical Tourism Pilot Zone. Raxone is the world’s first and only approved therapy for visual impairment in adolescent and adult patients with Leber’s Hereditary Optic Neuropathy (LHON).
Regulatory Milestone
| Item | Detail |
|---|---|
| Company | Chiesi Group (Italy‑based) |
| Drug | Raxone (idebenone 150 mg) |
| Approval | Boao Lecheng Pilot Zone (China) |
| Treatment Initiation | First patients at Ruijin Hainan Hospital |
| **Indication | Visual impairment in adolescent and adult LHON patients |
| **Mechanism | Antioxidant + mitochondrial electron transport chain cofactor |
| **Unique Position | World’s first and only approved LHON therapy |
Disease Profile: Leber’s Hereditary Optic Neuropathy (LHON)
- Etiology: Mitochondrial DNA mutations in Complex I (ND genes) affecting retinal ganglion cells (RGCs)
- Clinical Manifestations: Acute/subacute vision loss, typically in young adults; leads to central scotoma and legal blindness
- Epidemiology:
- Global prevalence: 1 in 30,000‑50,000
- China: ~3,500‑5,000 patients
- Onset: 90% male, typically aged 15‑35 years
- Unmet Need: No approved treatments until Raxone; disease causes irreversible vision loss within months
Drug Profile & Mechanism
- Dual Mechanism:
- Antioxidant: Scavenges free radicals, protecting RGCs from oxidative damage
- Mitochondrial Cofactor: Transfers electrons directly to Complex III, bypassing defective Complex I
- ATP Restoration: Restores cellular ATP production in dysfunctional RGCs
- Cellular Reactivation: Reactivates surviving but dysfunctional RGCs, leading to improved visual outcomes
- Administration: Oral tablets (150 mg), typically dosed three times daily
Market Opportunity & Competitive Landscape
| Parameter | China | Global |
|---|---|---|
| LHON Patients | 3,500‑5,000 | 60,000‑100,000 |
| Diagnosed & Eligible | 1,500 | 25,000 |
| Current Standard | No approved therapy; supportive care only | |
| Raxone Annual Cost (USD) | $25,000‑30,000 | $40,000‑50,000 |
| Market Value (2030E) | $45 million | $1.25 billion |
| Raxone Peak Share | 80% | 60% |
| Peak Revenue (2032E) | $36 million | $750 million |
Key Competitors:
- Gene Therapies: LUMEVOQ (GenSight Biologics) – EU approved, not yet in China; addresses only ND4 mutation
- Practical Vitamin Supplements – Unproven efficacy
- Raxone – Only approved therapy with global regulatory approval and broad LHON mutation coverage
Strategic Positioning
- China Market Access: Boao Pilot Zone approval enables early commercialization while NMPA review proceeds; Raxone eligible for NRDL fast‑track
- Manufacturing: Chiesi’s Parma, Italy facility supplies global markets; cold‑chain logistics established for China distribution
- Clinical Validation: >15 years of real‑world data from EU and US confirms visual acuity improvement in 30‑40% of patients
- Patient Support: Launching RaxONE Patient Program in China for access and adherence support
Forward‑Looking Statements
This brief contains forward‑looking statements regarding market penetration, revenue forecasts, and regulatory expansion for Raxone in China. Actual results may differ due to pricing negotiations, competitive gene therapies, and market access challenges.-Fineline Info & Tech