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Is Kebilidi available in the UAE? How families get Kebilidi (eladocagene exuparvovec-tneq)
A plain answer for families in the UAE, checked against FDA, manufacturer and regulator sources.
Reviewed by the Reserve Meds clinical operations team on 13 October 2026.
Is Kebilidi available in the UAE?
Kebilidi (eladocagene exuparvovec-tneq) is an FDA-approved one-time gene therapy given into the brain for AADC deficiency. As of 13 October 2026, we found no public record that Kebilidi is registered by EDE in the UAE. Kebilidi is given only at specialized treatment centers, so for most families in the UAE the realistic route is treatment at a qualified center abroad, after a referral from the treating specialist. If a hospital in the UAE wants to give it locally, the manufacturer must agree to supply that hospital and the Emirates Drug Establishment (EDE) must approve the import. Plan in months, not weeks (our planning estimate, not a promise). Reserve Meds helps organize the referral and logistics. We do not prescribe.
- Registration in the UAE: not found in public sources as of 13 October 2026. Your hospital confirms before filing.
- Legal route: Treatment at a qualified center abroad, or a hospital import request if the manufacturer agrees.
- Who applies: your treating specialist refers the patient to a qualified center; a hospital in the UAE applies to EDE only if the manufacturer agrees to supply it.
- Timeline: several months (not a promise).
- Medicine price: quote on request. Every case gets a written custom quote before you commit.
What Kebilidi is
Kebilidi (eladocagene exuparvovec-tneq) is made by PTC Therapeutics. The US FDA approved it on 13 November 2024 for adult and pediatric patients with aromatic L-amino acid decarboxylase (AADC) deficiency. It is given as a one-time infusion into a part of the brain called the putamen, given during a planned neurosurgical procedure.
The FDA decision was based on a trial that measured changes in motor milestones after 48 weeks. Because it is given by neurosurgery, the procedure itself carries surgical risks that the treating team will explain.
The same therapy was approved earlier in Europe under the name Upstaza, for a narrower group of patients, and in the UK, Israel, Brazil and Taiwan.
The legal route in the UAE
Kebilidi is given by a neurosurgical team at a specialized treatment center. It is not a medicine a family can collect or a clinic can give in an outpatient room.
For a family in the UAE, that leaves two realistic routes. First, treatment at a qualified center abroad, after your specialist refers you and the center accepts the patient. Second, a hospital in the UAE asks the manufacturer, PTC Therapeutics, whether it will supply that hospital. If the manufacturer agrees, the hospital still needs import approval from the Emirates Drug Establishment (EDE). We cannot promise that a manufacturer will agree.
How a hospital import request works in the UAE, if it is ever needed:
The Emirates Drug Establishment (EDE) is the UAE's federal medicines regulator. It was created by Federal Decree No. 28 of 2023 and takes over the medicines role of the Ministry of Health and Prevention (MOHAP). Medicines normally have to be registered before they are used.
For a medicine that is not registered, the treating hospital asks for approval to import it for a specific patient. The hospital signs an undertaking that it takes responsibility for the medicine's use, and it shows that the medicine is registered in its country of origin. FDA approval makes this easier. The medicine stays inside the hospital system and cannot be resold.
Hospitals in Abu Dhabi are licensed by the Department of Health (DoH), and hospitals in Dubai by the Dubai Health Authority (DHA). DoH publishes data showing that thousands of non-registered drug products have been used in Abu Dhabi under special authorization. Your hospital pharmacy will know which office handles the file for your hospital today.
Who does what
- Your treating specialist decides whether Kebilidi is right for the patient and writes the letter and prescription.
- The hospital refers the patient, or files a local import request if that becomes possible.
- The Emirates Drug Establishment (EDE) decides whether to approve an import of an unregistered medicine.
- PTC Therapeutics makes Kebilidi and decides where it can be supplied.
- Reserve Meds can help organize records and logistics, and keeps your family updated. We do not give medical advice.
Documents you will usually need
- A referral letter from the treating specialist: diagnosis, test results and why this treatment is being considered
- Genetic or laboratory reports that confirm the diagnosis, translated into English where needed
- Recent clinic notes, imaging and a list of past treatments
- Passport and travel documents, if treatment happens abroad
- A funding plan: personal funds, insurer approval, or a government treatment-abroad decision
Timeline, step by step
- Referral. Your specialist prepares a referral letter and the medical records.
- Center review. A qualified treatment center reviews the records and decides whether the patient is eligible.
- Funding and travel. The family confirms funding and plans travel and accommodation.
- Preparation. The center runs tests and, for some products, collects cells or tissue to make the treatment.
- Treatment and follow-up. The patient is treated and then followed up by the center and the home specialist.
The overall time is our planning estimate, not a promise. The regulator and the manufacturer set their own timelines.
What Kebilidi costs
Medicine price: quote on request. Your total cost depends on 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 shipping once approval is in place, and keep your family updated. We coordinate access to FDA-approved medicines only. We do not prescribe, and we do not decide what the regulator approves.
Ask about Kebilidi in UAE
Tell us the medicine and your country. We reply within one business day. On WhatsApp we usually reply the same day.
Frequently asked questions
Is Kebilidi approved in the UAE?
As of 13 October 2026, we found no public record that Kebilidi is registered by EDE in the UAE. The same therapy was approved earlier in Europe under the name Upstaza, for a narrower group of patients, and in the UK, Israel, Brazil and Taiwan. Ask your hospital pharmacy to confirm the current status with EDE and, for Abu Dhabi or Dubai hospitals, with DoH or DHA before anything is filed.
Who applies for permission to use Kebilidi in the UAE?
For treatment abroad, your specialist refers the patient to a qualified center. For a local hospital import, the hospital applies to the Emirates Drug Establishment (EDE), and the manufacturer must agree to supply.
How long does it take to get Kebilidi in the UAE?
Plan in months, not weeks. The center must review the records, accept the patient and prepare the treatment. This is our planning estimate, not a promise.
How much does Kebilidi cost?
Medicine price: quote on request. Your total cost, including shipping, import steps and coordination, is quoted in writing for your case before you commit. No hidden charges.
Can Kebilidi be given in the UAE instead of abroad?
Only if the manufacturer agrees to supply a hospital in the UAE and EDE approves the import. We will tell you honestly if that is not possible today.
Sources
Checked on 13 October 2026. Regulator rules change. Your hospital pharmacy confirms the current procedure before filing.
- FDA: KEBILIDI (eladocagene exuparvovec-tneq) product page, approved 13 Nov 2024
- Fierce Pharma: PTC wins FDA approval for first brain-delivered gene therapy Kebilidi, 14 Nov 2024
- AJMC: FDA approves gene therapy to treat AADC deficiency (administered into the putamen by neurosurgery)
- Baker McKenzie: new Emirates Drug Corporation replaces MOHAP as federal regulator of medical products (Federal Decree No. 28 of 2023)
- Al Tamimi & Co: importation of unregistered drugs in the UAE (hospital letter of undertaking; registration in country of origin)
- Department of Health Abu Dhabi: drug authorization dashboard (non-registered drug products with special authorization)