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Aqneursa (levacetylleucine): what it treats, how it is taken and how to access it

A plain overview for families, checked against the FDA label and Gulf regulator lists on 17 October 2026.

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

What is Aqneursa?

Aqneursa (levacetylleucine) is an oral medicine from IntraBio. The US FDA approved it for the neurological symptoms of Niemann-Pick disease type C in September 2024, and for ataxia in ataxia-telangiectasia in September 2026, in adults and children who weigh at least 15 kg. It has an active code in Abu Dhabi but is not in SFDA's list.

Uses (US label): neurological manifestations of Niemann-Pick disease type C; ataxia in ataxia-telangiectasia.
How it is taken: granules mixed into a drink, by mouth, up to three times a day, dosed by weight.
Gulf status: active code in Abu Dhabi; not found in SFDA's or NHRA's lists; Kuwait and Qatar not confirmed.
Timeline: We give you a written timeline estimate for your case before you commit.
Price: Medicine price: quote on request. You get a written quote for your case before you commit.

What Aqneursa treats

Aqneursa (levacetylleucine) is made by IntraBio. On 24 September 2024 the FDA approved it to treat the neurological manifestations of Niemann-Pick disease type C (NPC) in adults and in children who weigh at least 15 kg. The FDA gave it Priority Review, Fast Track, Orphan Drug and Rare Pediatric Disease designations.

In September 2026 the FDA added a second use: ataxia (loss of balance and coordination) in adults and children weighing at least 15 kg with ataxia-telangiectasia (A-T). It was described as the first FDA-approved treatment for A-T.

How it is taken

It comes as 1 gram of levacetylleucine granules in a unit-dose packet, mixed into a liquid and taken by mouth, up to three times a day, with or without food. The dose depends on body weight:

15 kg to under 25 kg: 1 g in the morning and 1 g in the evening
25 kg to under 35 kg: 1 g in the morning, 1 g in the afternoon and 1 g in the evening
35 kg or more: 2 g in the morning, 1 g in the afternoon and 1 g in the evening

Each packet is stirred into 40 mL of water, orange juice, almond milk or yogurt and taken within 30 minutes; hot liquids are not used. It can also be given through a G-tube of French size 12 or larger, mixed with water only. It is stored at room temperature (20°C to 25°C), so it does not need a cold chain.

The evidence behind the approvals

For NPC, the FDA reviewed a 24-week, randomized, double-blind, placebo-controlled crossover study in 60 patients aged 4 and older, with 12 weeks of treatment in each period. Miglustat was allowed as background treatment. Scores on a modified ataxia scale (fSARA) improved more with Aqneursa than with placebo.

For A-T, the phase 3 IB1001-303 crossover study enrolled 73 patients aged 4 to 50 with genetically confirmed A-T at 10 sites in Europe and the US. The ataxia score (SARA) improved by 1.9 points more with levacetylleucine than with placebo.

Safety

The US label warns that Aqneursa may harm an unborn baby. Women who could become pregnant should confirm they are not pregnant before starting and use effective contraception during treatment and for 7 days after the last dose. In the NPC study, the most common side effects were upper respiratory tract infection (17% versus 3% on placebo), abdominal pain, difficulty swallowing and vomiting (7% each). In the A-T study they were falls, skin cuts and urinary tract infections (6% each).

Status in the Gulf, with dates

Saudi Arabia: not found in SFDA's public Drugs List (all 9,173 entries searched by brand and generic name), checked 17 October 2026. (source)
UAE: active code QU4-D257-17316-01 in the Abu Dhabi Department of Health drug code list, release of 26 September 2026. Federal status is unverified. (source)
Bahrain: not on NHRA's Registered Medicine Price List (7 September 2026) or its Temporary Importation Medicines List (19 January 2025), checked 17 October 2026. (source)
Kuwait and Qatar: not confirmed, because we found no public, searchable drug register.

How families get Aqneursa

Where Aqneursa is listed, as in Abu Dhabi, a specialist can ask the hospital pharmacy whether it can be ordered through the local supply route. A drug code shows that a product is set up in that system; it does not confirm stock or funding.

Where it is not registered, the usual legal route is a named-patient import: the treating hospital asks the national regulator for permission to import it for one patient, using the FDA approval as the reference. In Saudi Arabia that is SFDA, in Bahrain NHRA, in Kuwait the Ministry of Health and in Qatar the Ministry of Public Health. Each regulator sets its own documents and conditions, which our country guides explain.

Because Aqneursa is stored at room temperature and taken by mouth, it does not need cold-chain shipping or an infusion center.

Country guides:

Documents you will usually need

A specialist's letter confirming the diagnosis: genetically confirmed NPC or A-T
The patient's current weight, which sets the dose
Recent neurological assessments, if available
For an import request: the hospital's request form, its signed declaration of responsibility, written patient or guardian consent, and proof of the US FDA approval (we can supply the US prescribing information)

Timeline and cost

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

Medicine price: quote on request. You get a written quote for your case before you commit. Your total cost depends on the dose, which follows body weight, plus 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 coordinate access to FDA-approved medicines only. We help your family understand the route in your country, organize records and communication with the hospital, and, where an import is approved, arrange US supply and shipping through our licensed wholesale partner. We do not prescribe, and we do not decide what a regulator approves.

Ask about Aqneursa

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 Aqneursa used for?

On the US label, Aqneursa treats the neurological manifestations of Niemann-Pick disease type C, and ataxia in ataxia-telangiectasia, in adults and children who weigh at least 15 kg.

Is Aqneursa available in the Gulf?

It has an active code in Abu Dhabi's drug code list (release of 26 September 2026). We did not find it in SFDA's public Drugs List in Saudi Arabia or on NHRA's lists in Bahrain, and Kuwait and Qatar have no public register we could check. Where it is not registered, the treating hospital can request a named-patient import.

How is Aqneursa taken?

By mouth, up to three times a day, with or without food. Each 1 g packet is mixed into 40 mL of water, orange juice, almond milk or yogurt. The dose depends on body weight.

How much does Aqneursa cost?

Medicine price: quote on request. You get a written quote for your case before you commit, including shipping, import steps and coordination. No hidden charges.

Does Aqneursa need refrigeration?

No. The US label says to store it at room temperature, 20°C to 25°C, with short excursions between 15°C and 30°C allowed.

Sources

Checked on 17 October 2026. Labels and regulator lists change. Your doctor or hospital pharmacy confirms the current position before anything is filed.