Actual patient not shown.

Act Today, Improve Tomorrow

AQNEURSA is the only FDA-approved stand-alone therapy for the treatment of Niemann-Pick disease type C (NPC)

In a clinical trial of 60 people with NPC, AQNEURSA improved neurological symptoms and functional abilities within 12 weeks compared with placebo.*

Talk to your healthcare provider about whether AQNEURSA could be right for you or someone you care for.

Discover more

FDA, US Food and Drug Administration.

*Neurological symptoms and functional outcomes were assessed using a modified version of the Scale for Assessment and Rating of Ataxia (SARA) called the functional SARA, or fSARA. If the fSARA score goes down, it means the symptoms measured are getting better overall. If the fSARA score goes up, it means the symptoms measured are getting worse. People with NPC who received AQNEURSA first followed by placebo had a greater improvement in the fSARA score with AQNEURSA. They had a mean change from baseline of -0.5 points with AQNEURSA, then 0 points with placebo. This was also seen in people who received placebo first followed by AQNEURSA. They had a mean change from baseline of -0.3 points with placebo, then -0.7 points with AQNEURSA.

Important Safety Information
Do not take AQNEURSA if you:
  • have not had a negative pregnancy test. If you become pregnant while taking AQNEURSA, inform your doctor immediately to discuss potential risks and alternative treatments
Before taking AQNEURSA, discuss with your doctor if you:
  • are pregnant or planning to become pregnant. Based on findings from animal reproduction studies, AQNEURSA may cause harm to your unborn baby when administered during pregnancy. If you become pregnant while taking AQNEURSA, inform your doctor immediately to discuss potential risks and alternative treatments.
  • require the need for effective contraception during treatment with AQNEURSA and for 7 days after stopping treatment to avoid pregnancy.
  • are breastfeeding or plan to breastfeed. It is not known if AQNEURSA or its metabolites pass into human or animal milk or effects on breastmilk production
  • are taking P-gp substrate. Your doctor will monitor you for related adverse reactions
  • have any other medical conditions
  • are taking any medications and supplements, including N-acetyl-DL-leucine or N-acetyl-D-leucine, as some of these may impact the effectiveness of AQNEURSA

The most common side effects of AQNEURSA include abdominal pain, dysphagia, upper respiratory tract infections and vomiting. Speak with your doctor if these side effects persist or worsen.

Take AQNEURSA exactly as prescribed by your doctor.

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.

AQNEURSA can be taken with or without food.

You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch, or call 1-800-FDA-1088 .

Back to top
Logo for IntraBio, a late-stage biopharmaceutical company focused on therapies for neurodegenerative diseases with high unmet medical needs

This site is intended for US residents only.

AQNEURSA, AQNEURSA Cares, and their respective logos are trademarks of IntraBio.

© 2025 IntraBio. All rights reserved. MAT-0053 01/25

Austin, TX

Contact Us
Privacy Policy
Cookie Policy

IMPORTANT SAFETY INFORMATION

Do not take AQNEURSA if you:

  • have not had a negative pregnancy test. If you become pregnant while taking AQNEURSA, inform your doctor immediately to discuss potential risks and alternative treatments

Before taking AQNEURSA, discuss with your doctor if you:

  • are pregnant or planning to become pregnant. Based on findings from animal reproduction studies, AQNEURSA may cause harm to your unborn baby when administered during pregnancy. If you become pregnant while taking AQNEURSA, inform your doctor immediately to discuss potential risks and alternative treatments.
  • require the need for effective contraception during treatment with AQNEURSA and for 7 days after stopping treatment to avoid pregnancy.
  • are breastfeeding or plan to breastfeed. It is not known if AQNEURSA or its metabolites pass into human or animal milk or effects on breastmilk production
  • are taking P-gp substrate. Your doctor will monitor you for related adverse reactions
  • have any other medical conditions
  • are taking any medications and supplements, including N-acetyl-DL-leucine or N-acetyl-D-leucine, as some of these may impact the effectiveness of AQNEURSA

The most common side effects of AQNEURSA include abdominal pain, dysphagia, upper respiratory tract infections and vomiting. Speak with your doctor if these side effects persist or worsen.

Take AQNEURSA exactly as prescribed by your doctor.

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.

AQNEURSA can be taken with or without food.

You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch, or call 1-800-FDA-1088 .

Indication

AQNEURSA™ (levacetylleucine) is indicated for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in adults and pediatric patients weighing ≥15 kg.

Please click here for Full Prescribing Information for AQNEURSA.