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Overview: Aqneursa is used to treat certain symptoms of Niemann-Pick disease type C or ataxia-telangiectasia, which are rare inherited conditions. It may help cells work better. Common side effects may include stomach pain, trouble swallowing, infection, and vomiting. If you can become pregnant, you should use effective birth control while taking Aqneursa and for 7 days after your last dose. Aqneursa comes as granules that are mixed in liquid before swallowing.

Uses

What is Aqneursa used for?

Aqneursa (levacetylleucine) is commonly used to help improve certain symptoms in people with the following rare genetic disorders.

Aqneursa may also be used for other conditions as determined by your health care provider.

How does Aqneursa work (mechanism of action)?

People with Niemann-Pick disease have a faulty gene that causes fat to build up in certain cells, which can lead to cell damage. People with ataxia-telangiectasia (A-T) have a faulty gene that affects their cells' ability to repair damaged DNA. In both conditions, Aqneursa may help cells work better, but exactly how it works is not yet known.

How is Aqneursa supplied (dosage forms)?

Brands and other names

  • Aqneursa

Dosage forms and strengths

  • 1-gram packet of granules for oral suspension

How should I store Aqneursa?

Aqneursa should be stored at room temperature, between 68 F and 77 F (20 C and 25 C). It can be exposed to temperatures between 59 F and 86 F (15 C and 30 C) for shorter periods of time, such as when transporting it. Store in a cool, dry place.

Side Effects

What are the most common side effects of Aqneursa?

The most common side effects of Aqneursa are listed below. These side effects may vary depending on the condition that is being treated. Tell your health care provider if you have any of these side effects that bother you.

  • Stomach pain
  • Trouble swallowing
  • Infection in the nose or throat, sore throat, or common cold symptoms
  • Vomiting
  • Falls
  • Skin tear
  • Kidney or bladder infection

There may be other side effects of Aqneursa that are not listed here. Contact your health care provider if you think you are having a side effect of a medicine. In the U.S., you can report side effects to the FDA at www.fda.gov/medwatch or by calling 800-FDA-1088 (800-332-1088). In Canada, you can report side effects to Health Canada at www.health.gc.ca/medeffect or by calling 866-234-2345.

What are the serious side effects of Aqneursa?

While less common, the most serious side effects of Aqneursa are described below, along with what to do if they happen.

Severe Allergic Reactions. Aqneursa may cause allergic reactions, which can be serious. Stop taking Aqneursa and get help right away if you have any of the following symptoms of a serious allergic reaction.

  • Breathing problems or wheezing
  • Racing heart
  • Fever or general ill feeling
  • Swollen lymph nodes
  • Swelling of the face, lips, mouth, tongue, or throat
  • Trouble swallowing or throat tightness
  • Itching or skin rash
  • Bumps on the skin called hives that can be red, pink, white, or brown depending on your skin tone
  • Nausea or vomiting
  • Dizziness, feeling lightheaded, or fainting
  • Stomach cramps
  • Joint pain

Warnings & Precautions

Who should not use Aqneursa?

Allergies to Ingredients. People who are allergic to any of the following should not take Aqneursa.

  • Aqneursa
  • Levacetylleucine
  • Any of the ingredients in the specific product dispensed

Your pharmacist can tell you all of the ingredients in Aqneursa.

What should I know about Aqneursa before using it?

Do not take Aqneursa unless it has been prescribed to you by a health care provider. Take it as prescribed.

Do not share Aqneursa with other people, even if they have the same condition as you. It may harm them.

Keep Aqneursa out of the reach of children.

Your dose of Aqneursa depends on how much you weigh. Keep all appointments so your health care provider can give you the right dose.

If you can become pregnant, your health care provider will likely do a test to be sure you are not pregnant before you start taking Aqneursa. You should also use effective birth control while taking Aqneursa and for 7 days after stopping it. See the Pregnancy section for more details.

Follow the “Instructions for Use” that come with Aqneursa for details on how to properly mix it before taking the medicine. Also follow these instructions for how to throw away any expired or unused Aqneursa that has already been mixed with liquid. Ask your pharmacist if you have any questions about these directions. 

What should I tell my health care provider before using Aqneursa?

Tell your health care provider about all of your health conditions and any prescription or over-the-counter (OTC) medicines, vitamins/minerals, herbal products, and other supplements you are using. This will help them know if Aqneursa is right for you.

In particular, make sure that you discuss any of the following.

Other Medicines and Supplements. Aqneursa may interact with other medicines and supplements. Before taking Aqneursa, tell your health care provider about any prescription or over-the-counter (OTC) medicines, vitamins/minerals, herbal products, and other supplements you are using. See the Interactions section for more details.

Pregnancy. Aqneursa may cause harm to a fetus. Tell your health care provider if you are pregnant, think you are pregnant, or plan to become pregnant.  

Breastfeeding. It is not known if Aqneursa passes into breast milk. Tell your health care provider if you are breastfeeding or plan to breastfeed. Your health care provider will let you know if you should take Aqneursa while breastfeeding.

How can I get more information about Aqneursa?

For more information about Aqneursa, you can visit the drugmaker’s website at aqneursa.com or call them at 866-200-0419.

Interactions

Does Aqneursa interact with foods or drinks?

There are no known interactions between Aqneursa and foods or drinks.

It is unknown if drinking alcohol will affect Aqneursa.

Does Aqneursa interact with other medicines (drug interactions)?

Always tell your health care provider about any prescription or over-the-counter (OTC) medicines, vitamins/minerals, herbal products, and other supplements you are using.

In particular, make sure that you discuss if you are using other acetylleucine products, such as N-acetyl-DL-leucine or N-acetyl-D-leucine, before taking Aqneursa.

Aqneursa may alter the blood levels of some other medicines. Tell your health care provider about all medicines that you take or have recently taken.

This may not be a complete list of medicines that can interact with Aqneursa. Always check with your health care provider.

Does interact with other drugs you are taking?

Enter your medication into the WebMD interaction checker

Overdose/Missed Dose

What should I do if I accidentally use too much Aqneursa?

If you or someone else has used too much Aqneursa, get medical help right away, call 911, or contact a Poison Control center at 800-222-1222.

What should I do if I miss a dose of Aqneursa?

If you miss a dose, skip the missed dose. Take the next dose when you normally would. Do not take double or extra doses.