Pronunciation: Ak-nur'-sah
Generic name: levacetylleucine
Dosage form: granules for oral suspension
Drug class: Miscellaneous metabolic agents

Medically reviewed by Carmen Pope, BPharm. Last updated on Sep 29, 2024.

What is Aqneursa?

Aqneursa (levacetylleucine) may be used to treat Niemann-Pick disease type C (NPC) in adults and children weighing 33 pounds ( ≥15 kg).

• Clinical trials have shown that Aqneursa can help improve both the neurological and functional symptoms of NPC, such as difficulty with walking, balance, speech, and fine motor skills within 12 weeks.

Aqneursa is a modified amino acid and its mechanism of action is thought to involve normalizing energy metabolism although the exact way it works is unknown.

Aqneursa gained FDA approval on September 24, 2024, and is the only FDA-approved stand-alone treatment that significantly improves NPC symptoms.

How do I get Aqneursa?

Aqneursa is not available at a retail or neighbourhood 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 see the Aqneursa Package Insert.

Aqneursa side effects

The most common side effects of Aqneursa are:

• abdominal (stomach) pain,
• difficulty swallowing
• colds and flu and other upper respiratory tract infections
• vomiting.

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

Serious side effects and warnings

Aqneursa may cause harm to an unborn baby. Based on findings from animal reproduction studies, Aqneursa may cause harm to your unborn baby when administered during pregnancy. Females who can become pregnant should use effective contraception while taking Aqneursa and for 7 days after finishing treatment.

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.

Related/similar drugs

Before taking

Do not take Aqneursa if you are a female who can get pregnant and you have not had a negative pregnancy test.

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 plan to breastfeed

Pregnancy

Aqneursa should not be taken during pregnancy. Your healthcare provider may ask you to conduct a pregnancy test before treatment which should be negative before starting treatment.

While you are taking Aqneursa you will need to use effective contraception during treatment with Aqneursa and for 7 days after stopping treatment to avoid pregnancy

If you become pregnant while taking Aqneursa, inform your doctor immediately to discuss potential risks and alternative treatments.

Breastfeeding

It is unknown whether Aqneursa or its metabolites pass into human or animal milk or affect breastmilk production.

How do I take Aqneursa?

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

Aquneursa 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 also be taken or given by mouth or through a gastrostomy tube (G-tube) feeding tube (French size 18 or larger).

How do I mix Aqneursa?

1. Open and empty the entire contents of one Aqneursa packet into a container with 40 mL of water, orange juice, or almond milk. Do not use hot liquid with Aqneursa oral suspension.
2. Stir to form a suspension.
3. Swallow the suspension immediately (within 30 minutes).
4. For doses requiring two Aqneursa packets, repeat the above steps.
5. Throw away unused Aqneursa suspension if not administered within 30 minutes (see disposal instructions below).

If administering Aqneursa via a gastrostomy feeding tube (G-Tube) (French size 18 or larger) prepare Aqneursa suspension immediately before administration.

• 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.
• Administer the suspension immediately through the G-tube.
• Flush any residual suspension in the catheter tip syringe with an additional 20 mL of water.
• Flush the G-tube again, as needed, until no residual suspension is left in the syringe or feeding tube.
• For doses requiring two Aqneursa packets, repeat steps 3 to 8.
• Discard unused Aqneursa suspension if not administered 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:

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

Aqneursa dosing information

The dose of Aqneursa depends on body weight.

What happens if I miss a dose of Aqneursa?

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.

What other drugs will affect Aqneursa?

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:

• A P-gp substrate, such as apixaban, colchicine, cyclosporine, dabigatran, digoxin, edoxaban, rivaroxaban, and tacrolimus with Aqneursa because it may increase the risk of side effects. Your healthcare provider will monitor you for these
• Are taking any medications and supplements that contain N-acetyl-DL-leucine or N-acetyl-D-leucine with Aqneursa, as some of these may affect the effectiveness of Aqneursa.

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.

Storage

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

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

Aqneursa ingredients

Active ingredients: levacetylleucine

Inactive ingredients: hypromellose, isomalt, and strawberry flavor.

One packet of Aqneursa for oral suspension contains 1 gram levacetylleucine as white to off-white strawberry flavored granules in a unit-dose packet to be mixed with water, orange juice, or almond milk before taking.

Who makes Aqneursa?

Aqneursa is made by IntraBio Inc.

Further information

Always consult your healthcare provider to ensure the information displayed on this page applies to your personal circumstances.

Medical Disclaimer