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Itvisma (onasemnogene abeparvovec-brve)

Creator: Reserve Meds
Published: 2026-10-17

A one-time gene therapy for spinal muscular atrophy, given into the spinal fluid. Facts checked against the US label and Gulf drug registers on 17 October 2026.

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

What is Itvisma?

Itvisma is a one-time gene therapy for spinal muscular atrophy (SMA). The US FDA approved it on 24 November 2025 for adults and children 2 years and older with a confirmed SMN1 gene mutation. It is given once into the spinal fluid by an experienced hospital team. Abu Dhabi lists it; SFDA's public list in Saudi Arabia does not.

US approval: 24 November 2025, Novartis Gene Therapies, Inc. (BLA 125856). Label revised June 2026. (source 3) (source 2)
Who it is for: adults and children 2 years and older with SMA and a confirmed SMN1 gene mutation.
How it is given: one injection into the spinal fluid (intrathecal), with steroid medicine before and after.
Gulf status: listed in Abu Dhabi; not found in SFDA's public list (Saudi Arabia) or NHRA's lists (Bahrain); other Gulf countries not confirmed.
Price: Quote on request.

What Itvisma is

Itvisma is an adeno-associated virus (AAV) vector-based gene therapy made by Novartis Gene Therapies, Inc. It carries a working copy of the gene that people with SMA are missing, and it is given only once. (source 2)

It has the same active substance as Zolgensma (onasemnogene abeparvovec), but it is a separate product with its own US approval, dose and route. Zolgensma is an intravenous infusion for children under 2. Itvisma is an injection into the spinal fluid for patients aged 2 and older. The US label says patients who were already treated with Zolgensma should not be treated with Itvisma, and Itvisma is not given a second time. (source 2)

Who it is for (FDA wording)

For the treatment of spinal muscular atrophy (SMA) in adult and pediatric patients 2 years of age and older with confirmed mutation in survival motor neuron 1 (SMN1) gene. (source 1)

In plain words: Itvisma is for children aged 2 and older, teenagers and adults with SMA whose genetic test confirms a mutation in the SMN1 gene. Whether it suits a particular patient is decided by the treating neurologist and the hospital.

Before treatment, the label asks the team to test for anti-AAV9 antibodies, check liver function, kidney function and blood counts, make sure vaccinations are up to date, and postpone treatment if the patient has an infection. (source 2)

How Itvisma is given

One dose, once. A fixed dose of 1.2 × 10¹⁴ vector genomes, injected into the spinal fluid by lumbar puncture over about 1 to 2 minutes.
By an experienced team. The label says it should only be given by healthcare professionals experienced in lumbar punctures, for example an interventional radiologist or neurologist.
Steroid medicine. Prednisolone (or an equivalent) starts one day before the injection and continues for 30 days. It is then tapered slowly and must not be stopped suddenly.
Monitoring. Liver tests continue for at least 3 months after the injection. Platelet counts are checked before treatment and at least weekly for the first month.
Frozen medicine. Itvisma is shipped and delivered frozen (at or below minus 60°C) in a single-dose vial and is thawed before use. (source 2)

Because of the frozen supply, the procedure and the follow-up, Itvisma is a hospital treatment. It is not a medicine a family collects or carries home. Planning starts with the hospital that will give it.

What the main study showed

The US label describes a randomized, sham-controlled study of 126 treated patients aged 2 to 17 who had not had SMA treatment before, and who could sit but had never walked on their own. After about one year (average of weeks 48 and 52), the motor score (HFMSE, maximum 66) rose by 2.39 points with Itvisma and by 0.51 points with the sham procedure, a difference of 1.88 points. (source 2)

Safety: what the label warns about

Itvisma has a boxed warning for serious liver injury. Patients with existing liver problems may be at higher risk. The label also warns about low platelet counts, nerve problems in the hands and feet (sensory neuropathy), thrombotic microangiopathy (a rare blood clotting problem that can harm the kidneys), raised cardiac troponin I, and a theoretical risk of tumors from the gene vector. The most common side effects in the studies were upper respiratory infections, stomach upset, fever and headache. (source 2)

Itvisma in the Gulf: register status

We checked the public registers below on 17 October 2026. A listing shows that the product is set up in that system. It does not tell you about stock, eligibility or funding.

Saudi Arabia: not found in SFDA's public Drugs List (all 9,173 entries searched by brand and generic name). Zolgensma, the intravenous product, is listed. (source 4)
UAE, Abu Dhabi: ITVISMA is in the Department of Health drug code list (release 26 September 2026) with two codes, one active and one in grace status. The federal EDE and Dubai DHA status were not checked. (source 5)
Bahrain: not found in NHRA's Registered Medicine Price List (7 September 2026) or its Temporary Importation Medicines List (19 January 2025). (source 6) (source 7)
Kuwait, Qatar and Oman: we did not find a public searchable register, so the status is not confirmed.

Public lists can lag behind decisions, so the hospital pharmacy confirms the current status with the regulator before anything is filed.

How access works

Where it is listed (Abu Dhabi). Start by asking a qualified hospital's neuromuscular team whether it can order Itvisma through the local supply route.

Where it is not registered. Because Itvisma is FDA-approved, the treating hospital can ask the national regulator for permission to import it for one named patient. In Saudi Arabia the SFDA Special Access Program guidance covers this; in Bahrain, NHRA Circular 41 of 2020 sets the procedure. The hospital pharmacy knows which route fits the case. (source 8) (source 9)

Treatment abroad. If no local hospital can give it, the treating specialist can refer the patient to a qualified hospital in another country.

Who does what

Your treating neurologist decides whether Itvisma is right for the patient and writes the medical letter.
The hospital checks eligibility, files any import request, receives the frozen medicine and is responsible for giving it safely and for follow-up.
The regulator (for example SFDA, DoH Abu Dhabi or NHRA) decides on registration and on any import request.
Novartis Gene Therapies makes Itvisma and decides where it can be supplied.
Reserve Meds helps your family understand the steps, organizes records and communication, and, where an import is approved, arranges US supply through our licensed wholesale partner. We do not give medical advice.

Documents you will usually need

The genetic report that confirms the SMN1 mutation
A letter from the treating neurologist with the diagnosis, past and current SMA treatments (including whether Zolgensma was ever given) and why Itvisma is being considered
Recent clinic notes and laboratory results
Identity documents and a funding plan (personal funds, insurer approval or a government treatment decision)
Passport and travel documents, if treatment happens abroad

Timeline and cost

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

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

Country guides for Itvisma

How Reserve Meds helps

Reserve Meds is a digital health company. Medicines are supplied through a US-licensed pharmaceutical wholesale partner. We coordinate access to FDA-approved medicines only. We help your family understand the route, organize records and communication with the hospital, and, where an import is approved, arrange US supply and shipping. We do not prescribe, and we do not decide what the regulator or a hospital approves.

Ask about Itvisma

Tell us the medicine, your country and a WhatsApp or phone number. We reply within one business day. On WhatsApp we usually reply the same day.

Frequently asked questions

What is Itvisma used for?

Itvisma is a one-time gene therapy for spinal muscular atrophy (SMA). The US FDA approved it on 24 November 2025 for adults and children 2 years of age and older with a confirmed mutation in the SMN1 gene.

How is Itvisma different from Zolgensma?

Both contain onasemnogene abeparvovec and are made by Novartis Gene Therapies, but they are separate products. Zolgensma is an intravenous infusion for children under 2. Itvisma is a single injection into the spinal fluid for patients 2 and older. The US label says patients already treated with Zolgensma should not be given Itvisma.

Is Itvisma available in Saudi Arabia or the UAE?

On 17 October 2026, Itvisma was listed in the Abu Dhabi Department of Health drug code list with an active code. We did not find it in SFDA's public Drugs List in Saudi Arabia, or in Bahrain's NHRA lists. Status in Kuwait, Qatar, Oman and at the UAE federal level is not confirmed. Where it is not registered, the treating hospital can ask the regulator about a named-patient import.

How is Itvisma given?

It is given once, as an injection into the spinal fluid by lumbar puncture, by a team experienced in that procedure. Steroid medicine starts one day before and continues for 30 days, then is tapered. Liver tests continue for at least 3 months. The medicine is shipped frozen, so it is a hospital treatment, not a medicine a family collects.

How much does Itvisma cost?

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

Can Reserve Meds decide if my child is eligible for Itvisma?

No. Your treating neurologist and the hospital decide if Itvisma is right for the patient. Reserve Meds is a digital health company. We help your family understand the route, organize records and, where an import is approved, arrange US supply through our licensed wholesale partner. We do not prescribe.

Sources

Checked on 17 October 2026. Regulator rules and lists change. Your hospital pharmacy or treatment center confirms the current position before anything is filed.

This page is general information, not medical advice. Read the full US prescribing information and talk to your own doctor.