Example scenario: a family in Riyadh asks about a newly approved SMA gene therapy

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

The situation

A family in Riyadh has a 4-year-old son with spinal muscular atrophy (SMA). A genetic test confirmed a mutation in his SMN1 gene. He can sit on his own but has never walked.

His neurologist has read about Itvisma (onasemnogene abeparvovec-brve), a one-time gene therapy given into the spinal fluid. The US FDA approved it on 24 November 2025 for SMA in adults and children aged 2 and older with a confirmed SMN1 mutation. In the main study in the US label, 126 patients aged 2 to 17 who could sit but had never walked received Itvisma or a sham procedure.

The family asks three questions: can he get it in Saudi Arabia, what is needed, and how long will it take?

Why it is not simple

Itvisma is not in the Saudi Food and Drug Authority (SFDA) public Drugs List. We checked all 9,173 entries by brand and generic name on 17 October 2026.

SFDA registered Zolgensma, the intravenous gene therapy with the same active substance, in May 2022. Itvisma is a separate product, so it needs its own registration or a permission to import it for this patient.

What happens, step by step

1. The neurologist decides and explains.

   Only the treating specialist can decide whether Itvisma is right for this child. SFDA's Special Access Program guidance says registered alternatives should always be considered first, so the letter explains why Itvisma is being requested instead of a registered SMA treatment.

   The same guidance makes the treating physician responsible for explaining the possible risks, benefits and alternatives, and for getting informed consent from the patient or the legal guardian. Here, the parents sign.

2. Baseline checks before any request.

   The US label lists checks before the injection: any infection must have cleared, vaccinations should be up to date, and the team tests liver function, kidney function (creatinine), a full blood count including platelets, and antibodies to AAV9, the virus shell that carries the gene.

   Itvisma has a boxed warning for serious liver injury, which is why the liver tests matter so much.

3. The hospital chooses the SFDA route and files.

   A licensed Saudi hospital files the request, not the family. In the named-patient route, the medicine must be approved by a reference regulator such as the US FDA, the hospital signs a declaration of responsibility for safe use, and a licensed importer signs a declaration covering quality during transport.

   SFDA also runs the Special Access Program, whose guidance names the product's sponsor, or the licensed treating physician when there is no sponsor, as the requester. The hospital pharmacy knows which route fits this case. Reserve Meds does not file on the family's behalf.

4. SFDA reviews the request.

   The Special Access Program guidance says a complete request is processed within 24 working hours and then passed to an assessor. It does not set a fixed time for the assessor's review, because each request is judged on its own facts. SFDA can approve, ask the doctor for more information, or refuse.

   The department's working hours are 07:30 to 15:30, Sunday to Thursday. A request only starts moving once it is complete, so the hospital checks the document list before it submits.

5. Supply and booking the injection date.

   After approval, Reserve Meds arranges supply through our US-licensed pharmaceutical wholesale partner to the hospital pharmacy named in the approval, where the product can be sourced. Nothing goes to the family's home.

   The US label says Itvisma is shipped frozen at minus 60 degrees Celsius or colder. On arrival it goes into a refrigerator at 2 to 8 degrees Celsius and must be used within 14 days. So the hospital books the injection date before the shipment leaves.

6. Injection day and the months after.

   Steroid medicine starts one day before the injection, at 1 mg per kg of body weight per day, for 30 days. If the liver tests are reassuring, the dose is then reduced gradually over at least 28 days. Steroids must not be stopped suddenly.

   Liver tests are done every week in the first month and during the taper, then every other week for another month if results are normal. Platelets are checked weekly for the first month. Liver monitoring continues for at least 3 months in total. The family should plan around this follow-up.

Which time points are fixed

Regulators, manufacturers and hospitals set their own timelines, and most do not publish a fixed review time. We give you a written timeline estimate once we see the hospital file.

Documents usually needed

• A letter from the treating neurologist: diagnosis, current and past SMA treatments, and why Itvisma is requested instead of a registered alternative
• The genetic report that confirms the SMN1 mutation
• Recent clinic notes and motor assessments
• Baseline test results: liver and kidney function, full blood count, AAV9 antibody test, vaccination record
• The hospital's request and its signed declaration of responsibility, plus the importer's declaration
• Written consent signed by the parents as legal guardians
• Proof of the US FDA approval and the US prescribing information (we can supply these)

What it may cost

Quote on request. Hospital charges for the injection and follow-up are set by the hospital. Your coordination fee is quoted in writing for your case before you commit. No hidden charges.

What Reserve Meds does, and does not do

Reserve Meds is a digital health company. Medicines are supplied through a US-licensed pharmaceutical wholesale partner. Reserve Meds can explain the route, help the doctor and hospital assemble the file, arrange US supply through our wholesale partner after approval, coordinate cold-chain shipping, and keep the family updated. We do not prescribe, we do not decide eligibility, and we do not decide what SFDA approves.

Related

• Itvisma in Saudi Arabia: access guide
• Saudi Arabia country guide
• Named-patient access in the Gulf, explained
• All example scenarios
• Our editorial standards
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This page is general information, not medical advice. Your own doctor decides what is right for you.