Leber congenital amaurosis (LCA) and RPE65-related inherited retinal dystrophy: US-approved treatments and how families in the Gulf can access them

Reviewed by the Reserve Meds clinical operations team on 17 October 2026.

What is Leber congenital amaurosis and RPE65 retinal dystrophy?

Leber congenital amaurosis and early-onset severe retinal dystrophy are inherited diseases that cause severe vision loss early in life. Leber congenital amaurosis usually refers to vision loss in the first year of life, and early-onset severe retinal dystrophy to vision loss between ages 1 and 5.^[1]

These disorders mainly affect the retina, the tissue at the back of the eye that detects light and color. There are many types, depending on the gene involved, and vision may worsen over time.^[1]

Leber congenital amaurosis occurs in two to three in 100,000 newborns and is among the leading genetic causes of severe vision loss in infants. For people with changes in both copies of the RPE65 gene, a one-time gene therapy is approved in the US.^{[1, 2]}

Leber congenital amaurosis and RPE65 retinal dystrophy in the Gulf

Gulf genetic eye research includes a 2025 study exploring the genetic causes of nonsyndromic retinal dystrophies in Qatar and a 2016 study of a novel GUCY2D mutation causing Leber congenital amaurosis in a large kindred.^{[3, 4]}

We checked every medicine on this page against the full SFDA drugs list and the Abu Dhabi Department of Health drug code list on 17 October 2026. 1 of 1 was listed in Saudi Arabia and 1 of 1 in Abu Dhabi. The result for each medicine is shown below. A listing does not mean every hospital stocks the medicine, and federal UAE, Kuwait, Qatar, Bahrain and Oman registers were not checked.^{[5, 6]}

US FDA-approved medicines for Leber congenital amaurosis and RPE65 retinal dystrophy

Each medicine below is approved in the US for a specific group of patients. The wording summarizes the current US label. Your specialist decides whether any of them is right for the patient.

• Luxturna (voretigene neparvovec-rzyl)
  US FDA-approved for patients with confirmed biallelic RPE65 mutation-associated retinal dystrophy who have enough viable retinal cells. It is a one-time gene therapy injected under the retina during eye surgery at a qualified center.^[2]
  Current US label version: 1 May 2022. Gulf registers: Saudi Arabia (SFDA drugs list): listed. Abu Dhabi (DoH drug code list): listed. Checked 17 October 2026. Federal UAE, Kuwait, Qatar, Bahrain and Oman: not checked; we confirm for your country when you ask.^{[5, 6]}
  Country access guides: Saudi Arabia, UAE, India

How families in the Gulf can access treatment

If the medicine is listed in your country. Ask your specialist and the hospital about local supply first. A listing does not mean every hospital stocks the medicine, but it is usually the fastest route, and you may not need us.

If it is not registered. The usual legal route is a named-patient request: the treating hospital, on your specialist's written request, asks the national regulator for permission to import the medicine for one patient, using the US FDA approval as the reference. In Saudi Arabia the SFDA Special Access Program guidance covers these requests; in Bahrain, NHRA Circular 41 of 2020 sets the procedure. Other Gulf countries have their own procedures, which the hospital confirms.^{[7, 8]} Once approval is in place, Reserve Meds arranges supply through a US-licensed pharmaceutical wholesale partner to the hospital.

For Luxturna. These are made for one patient or given through a specialized procedure, so treatment happens at a qualified center. For most Gulf families that means a referral from the treating specialist to a center that can give the treatment, which may be abroad. A local hospital can only give it if the manufacturer agrees to supply that hospital and the regulator approves. We cannot promise that a manufacturer will agree.

Who does what

• Your treating specialist decides whether a medicine is right for the patient and writes the medical letter.
• The hospital checks local supply, files any import request, and gives the treatment safely.
• The national regulator decides whether to approve the import of a medicine that is not registered.
• The manufacturer decides where its medicine can be supplied, especially for cell and gene therapies.
• Reserve Meds helps organize records, coordinates US supply through our wholesale partner, shipping and updates to your family. We do not give medical advice.

Documents you will usually need

• A letter from the treating specialist with the diagnosis, past treatments and why this medicine is being considered
• Reports that confirm the diagnosis, such as genetic, pathology, imaging or laboratory results, translated into English where needed
• Recent clinic notes and a list of current medicines
• For a hospital import request: the prescription, the hospital's request form and the written consent of the patient or guardian
• Proof of the US FDA approval and the US prescribing information (we can supply these)

We give you a written timeline estimate for your case before you commit. The regulator, the hospital or treatment center and the manufacturer set their own timelines.

What treatment costs

Quote on request. Your total cost depends on the medicine, the dose, shipping, import steps and coordination. Your coordination fee is quoted in writing for your case before you commit. No hidden charges.

How Reserve Meds helps

Reserve Meds is a digital health company. Medicines are supplied through a US-licensed pharmaceutical wholesale partner. We help your doctor and hospital prepare the file, arrange US supply and temperature-controlled shipping once approval is in place, and keep your family updated. We coordinate access to FDA-approved medicines only. Investigational medicines are only available through a manufacturer's expanded-access program requested by the treating physician; we can explain that route, but we do not supply them.

Sources for the condition-specific answers: ^[2]

Related

• Keratoconus
• Glaucoma
• Luxturna: drug overview
• Named-patient access in the Gulf, explained
• US-approved medicines not yet registered in the Gulf
• Answers to common access questions
• Cross-border disclosures
• اقرأ هذه الصفحة بالعربية

This page is general health information, not medical advice. It does not replace a consultation with your doctor.