Updated: Oct 19
Medically reviewed by ChinaCureLink Team
Table of Content
Why This Case Matters
This case follows the rollout of Raxone (idebenone) for Leber Hereditary Optic Neuropathy at a hospital within Hainan's Boao Lecheng Pilot Zone — a treatment that reached 100 patients in just seven months after becoming accessible in mainland China for the first time.
For international patients with LHON, including those in Southeast Asia, where this pathway has already reached patients from Myanmar, ChinaCureLink can help evaluate and coordinate access to this same treatment.
Key Takeaways |
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Background
LHON is a maternally inherited mitochondrial disease that typically strikes adolescents and young adults, causing sudden, painless, and severe vision loss in both eyes. It's listed in China's first national catalog of rare diseases. For years, Chinese LHON patients faced a stark reality: no approved treatment was available to them in mainland China, even though an effective therapy already existed elsewhere in the world. The hospital's 100th patient under this pathway, from Anhui province, was one of many who had been living with this diagnosis with no treatment option available domestically until now.
The Care Team and Approach
Raxone (idebenone 150mg), developed by Italy's Chiesi Group, is the world's first and only drug approved specifically to treat visual impairment in adolescent and adult LHON patients. It has held marketing approval in the European Union since 2015, along with the UK, Israel, South Korea, Switzerland, and several other countries, and its efficacy and safety have been supported by international clinical studies and multiple authoritative clinical consensus statements.
Despite this international track record, Raxone had never been available to patients in mainland China — until Hainan's Boao Lecheng Pilot Zone, operating under a special "first-to-try" regulatory policy, approved the drug for use at a hospital within the zone in December 2025, following close coordination between Hainan's provincial drug administration, health commission, customs authority, and the Lecheng zone's own medical products administration. The hospital's specialist team quickly established a standardized diagnosis and treatment protocol to ensure patients received safe, consistent care, treating its first 6 patients on January 17, 2026.
Treatment Timeline/ Process
Before This Pathway
LHON patients across China lived with progressive vision loss with no approved treatment available domestically, despite Raxone being approved internationally since 2015
At Hainan's Boao Lecheng Pilot Zone
December 2025: Raxone officially approved for use at the hospital under Lecheng's special access policy
January 17, 2026: First group of 6 patients began treatment
Over the following 7 months: Patient numbers grew steadily, reaching individuals across 26 Chinese provinces and regions, plus international patients from Japan and Myanmar
August 28, 2026: The hospital's 100th patient, from Anhui province, completed treatment
Outcome
Within seven months of its regional debut, Raxone had reached 100 LHON patients at the hospital — a pace the treating institutions have described as demonstrating both the speed of Lecheng's regulatory pathway and the strength of its clinical system integration. Individual visual outcomes vary by case and disease stage, but Raxone's efficacy in improving visual acuity for LHON patients has been supported by international clinical trial data, and the treatment protocol established at the hospital continues to expand access to patients across China and the surrounding region.
How ChinaCureLink Can Help International Patients to Access This Treatment
Two of the first 100 patients treated through this pathway already traveled internationally — from Japan and Myanmar — demonstrating that this access route is genuinely open to patients outside mainland China, not just domestic cases. For international patients with LHON, particularly across Southeast Asia, where this precedent already exists, ChinaCureLink can help coordinate a case review and connect patients with the hospital's specialist team, managing medical record translation, treatment scheduling, and logistics from first inquiry through treatment.
Get a Second Opinion Before Fly to China
For suitable cases, ChinaCureLink can then coordinate an online consultation with the specialist to discuss the technical treatment details directly. The purpose is to help the patient understand which treatment option and which specialist center may be most appropriate before making an international treatment decision.
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Frequently Asked Questions
Is Raxone (idebenone) available for LHON treatment in mainland China?
Raxone is not broadly approved in mainland China, but it is accessible through Hainan's Boao Lecheng Pilot Zone under a special early-access regulatory pathway, at a hospital specifically approved to administer it.
Can international patients access Raxone treatment at Hainan's Boao Lecheng zone?
Yes — international patients, including those from Japan and Myanmar, have already accessed this treatment through the pathway. ChinaCureLink can help coordinate a case review to evaluate eligibility.
How effective is Raxone for treating vision loss from LHON?
Raxone is the only drug specifically approved to treat visual impairment in adolescent and adult LHON patients, with efficacy supported by international clinical studies. Individual results vary depending on disease stage and other clinical factors, so a specialist evaluation is recommended.
About ChinaCureLink
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Disclaimer
This account reflects a treatment program's reported outcomes, which will vary by individual case, diagnosis stage, and other clinical factors. ChinaCureLink facilitates access to hospitals and treatment coordination — we do not provide medical care ourselves, and this content is not medical advice. Patients and families should consult a qualified ophthalmologist or geneticist before making treatment decisions.
