Aqneursa

Aqneursa (levacetylleucine) is used in adults and children weighing 33 pounds (15 kg) or more to treat:

• neurological symptoms of Niemann-Pick disease type C (NPC)
• ataxia in those with ataxia-telangiectasia (A-T). Ataxia can include problems with walking, balance, speech, eye movements, and hand coordination.

Aqneursa is given by mouth or through a gastrostomy tube (G-tube), 2 to 3 times a day depending on body weight.

In clinical trials, Aqneursa improved neurological function, including walking, sitting, standing, and speech, compared with placebo after 12 weeks of treatment in people with NPC and in people with A-T.

Aqneursa contains levacetylleucine, a modified amino acid. The exact way it works in NPC or A-T is not known.

Aqneursa (levacetylleucine) was first approved by the FDA on September 24, 2024, for NPC. Approval was extended on September 18, 2026, for ataxia in A-T, the first treatment approved for ataxia in patients with A-T.

Aqneursa led to statistically significant improvements compared with placebo in two clinical trials, one in people with NPC and one in people with A-T. In both trials, each patient took Aqneursa for 12 weeks and placebo for 12 weeks, in random order.

Effectiveness was measured using the Functional Scale for Assessment and Rating of Ataxia (fSARA), which scores walking, sitting, standing, and speech from 0 to 16. A lower score means better function.

In the NPC trial, most patients (85%) were also taking miglustat.

Aqneursa is not available at a retail or neighborhood pharmacy. Aqneursa is distributed by Curant Health, a rare disease-experienced specialty pharmacy.

To receive access to Aqneursa, have your doctor visit www.aqneursa.com and complete the enrollment form. Your healthcare provider can write you a prescription for Aqneursa.

Aqneursa Cares program

Patients with commercial insurance may be eligible for assistance with their co-pay for Aqneursa. If you are unable to afford your co-pay and you have commercial insurance (employer-sponsored or individual), you may be eligible for co-pay assistance where allowed by law. For more information, visit www.aqneursa.com or call Aqneursa Cares at 866-200-0419.

Take or give Aqneursa oral suspension exactly as instructed by your healthcare provider.

Aqneursa is taken two to three times per day, depending on body weight.

• Take or give Aqneursa oral suspension with or without food.
• Aqneursa oral suspension can be taken or given by mouth or through a gastrostomy tube (G-tube) feeding tube (French size 12 or larger).
• Do not use hot liquid with Aqneursa oral suspension.

How do I mix Aqneursa?

Taking Aqneursa by mouth

• Get the number of Aqneursa packets needed for your prescribed dose (one or two packets).
• Open and empty the entire contents of one Aqneursa packet into a container with 40 mL of water, orange juice, almond milk, or yogurt. Do not use hot liquid.
• Stir to form a suspension.
• Swallow the suspension immediately (within 30 minutes).
• For doses requiring two Aqneursa packets, repeat steps 2 to 4.
• Throw away unused Aqneursa suspension if not taken within 30 minutes (see disposal instructions below).

Giving Aqneursa through a gastrostomy tube (G-tube)

Use a G-tube that is French size 12 or larger.

• Prepare Aqneursa suspension immediately before giving it through the G-tube.
• Get the number of Aqneursa packets needed for your prescribed dose (one or two packets).
• Open and empty the entire contents of one Aqneursa packet into a container with 40 mL of water ONLY. Do not use hot liquid.
• Stir to form a suspension.
• Draw up the suspension into a catheter-tip syringe.
• Give the suspension immediately through the G-tube.
• Flush any remaining suspension in the catheter tip syringe with an additional 20 mL of water.
• Flush the G-tube again, as needed, until no suspension is left in the syringe or feeding tube.
• For doses requiring two Aqneursa packets, repeat steps 3 to 8.
• Throw away unused Aqneursa suspension if not given immediately (see disposal instructions below).

How to dispose of expired Aqneursa packets or unused Aqneursa oral suspension

Throw away (dispose of) expired Aqneursa packets or unused Aqneursa oral suspension by following the steps below:

• Mix the medicine with a substance such as dirt, cat litter, or used coffee grounds.
• Place the mixture in a container such as a sealed plastic bag.
• Throw away (dispose of) the container in your household trash.

Before taking Aqneursa, tell your doctor about all your medical conditions, including if you are:

• taking any other medications or supplements
• pregnant or planning to become pregnant
• breastfeeding or planning to breastfeed.

Pregnancy

Aqneursa may cause harm to an unborn baby. If you can become pregnant, your healthcare provider will check that you are not pregnant before you start treatment.

Use effective contraception during treatment with Aqneursa and for 7 days after the last dose if Aqneursa is stopped.

If you become pregnant while taking Aqneursa, tell your healthcare provider right away. Your healthcare provider will help you decide whether to continue Aqneursa, taking into account your need for treatment, the possible risks to your baby, and the risks of your condition going untreated.

Breastfeeding

It is not known whether levacetylleucine or its metabolites pass into breast milk or affect breast milk production. Talk to your healthcare provider about the benefits of breastfeeding and your need for Aqneursa.

The most common side effects of Aqneursa in people with NPC are:

• abdominal (stomach) pain
• difficulty swallowing
• upper respiratory tract infections, such as colds
• vomiting.

The most common side effects of Aqneursa in people with A-T are:

• falls
• skin cuts or tears (skin laceration)
• urinary tract infections.

Speak with your doctor if these side effects persist or worsen.

How common are Aqneursa side effects?

In the NPC trial, one patient had a flare of rosacea that responded to treatment, and Aqneursa was continued. Four patients had low platelet counts (thrombocytopenia); all were also taking miglustat, and in two of them the low platelet count was present before starting Aqneursa.

Aqneursa may cause harm to an unborn baby. Based on findings from animal reproduction studies, Aqneursa may cause harm to your unborn baby when taken during pregnancy. If you can become pregnant, your healthcare provider will check that you are not pregnant before you start Aqneursa. Use effective contraception during treatment with Aqneursa and for 7 days after the last dose if Aqneursa is stopped. Tell your healthcare provider right away if you become pregnant or think you may be pregnant.

Report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch, or call 1-800-FDA-1088. See the Aqneursa Prescribing Information for a complete list of side effects.

The dose of Aqneursa depends on body weight. One Aqneursa packet contains 1 gram of levacetylleucine. For 2-gram doses, prepare each packet separately.

If a dose of Aqneursa is missed, skip the missed dose and take the next dose at the scheduled time. Do not take 2 doses at the same time to make up for a missed dose.

Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. Especially tell your healthcare provider if you take:

• N-acetyl-DL-leucine or N-acetyl-D-leucine. Avoid taking these with Aqneursa, because they may make Aqneursa less effective.
• Medicines that are P-glycoprotein (P-gp) substrates. Aqneursa may increase the risk of side effects from these medicines, so your healthcare provider may monitor you more closely.

Other drugs may interact with Aqneursa, including prescription and over-the-counter medicines, vitamins, and herbal products. Not all possible drug interactions are listed here.

Store Aqneursa at room temperature between 68°F and 77°F (20°C and 25°C).

Keep Aqneursa oral suspension and all medicines out of reach of children.

Active ingredient: levacetylleucine

Inactive ingredients: hypromellose, isomalt, and strawberry flavor.

Aqneursa is supplied as white to off-white, strawberry-flavored granules in unit-dose packets, each containing 1 gram of levacetylleucine, in cartons of 28 packets.

Aqneursa is distributed by IntraBio Inc., Austin, TX 78701, and is a trademark of IntraBio.