Leber hereditary optic neuropathy (LHON) and inherited retinal disease: cross-border specialty drug access for international patients

Quick orientation

LHON affects an estimated 1 in 30,000 to 50,000 people, with male predominance. Inherited retinal diseases collectively affect approximately 1 in 3,000 people globally.

Typical age of onset. LHON most commonly between ages 15 and 35; other inherited retinal diseases vary widely in onset.

Severity tiers. LHON typically causes severe central vision loss within months of onset. Other inherited retinal diseases range from slowly progressive night blindness to rapid pediatric blindness.

Why specialty drugs for Leber hereditary optic neuropathy (LHON) and inherited retinal disease are hard to access internationally

Luxturna is the only FDA-approved gene therapy for inherited retinal disease (RPE65-mediated), administered subretinally at qualified centers. Other inherited retinal diseases including LHON have limited targeted therapy options, and disease-specific gene therapies are in development.

Treatments approved by the FDA

• Luxturna (voretigene neparvovec) — FDA approval: 2017. Mechanism: Subretinal AAV2-vectored gene therapy delivering RPE65. Route: One-time subretinal injection per eye. US WAC ballpark: Approximately USD 425,000 per eye. Country pricing: UAE · Saudi Arabia · India.

Cross-border pathways used for Leber hereditary optic neuropathy (LHON) and inherited retinal disease

Most patients use one or more of the following regulatory pathways, depending on the destination country and the specific drug:

• Medical Tourism Access
• Named Patient Program (NPP)
• Expanded Access Program (EAP)
• Compassionate Use

What your physician needs to know

• Confirm RPE65 biallelic pathogenic variants for Luxturna eligibility.
• Document baseline visual function and viable retinal cells on OCT.
• Pediatric eligibility: 12 months and older per label.
• Treatment is administered at qualified ophthalmic surgical centers.
• Coordinate with inherited retinal disease specialist.

Common questions

Is Luxturna available in my country?

Most administrations occur at qualified centers in the US and EU. Reserve Meds supports medical-tourism access.

How is it administered?

Surgical subretinal injection by a trained vitreoretinal surgeon.

What about other inherited retinal diseases?

Targeted therapy options are limited but expanding. Several gene therapies are in late-stage development.

Who pays for medical tourism?

Reserve Meds operates cash-pay. Some families combine personal funds with foundation support.

What documents are required?

Genetic confirmation, ophthalmologic workup, and treating specialist's referral.

Where Reserve Meds fits in

Reserve Meds is a cross-border specialty drug access platform. We support international patients whose prescribed FDA-approved medicine is not registered locally, is not reimbursed by their payer, or is otherwise unavailable through standard channels. For Leber hereditary optic neuropathy (LHON) and inherited retinal disease, our role is to coordinate the regulatory pathway, source the medicine from a DSCSA-compliant US wholesaler, and arrange validated cold-chain or controlled-temperature shipment to the destination country.

We do not replace your treating physician. We do not bill insurance. We operate a cash-pay model, and we work alongside the clinical team that knows your case. Every prescription is reviewed by a US-licensed pharmacist before dispense, and a US-licensed physician reviews the supply request before shipment.

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