AQNEURSA® (levacetylleucine) is now approved for the treatment of Ataxia-Telangiectasia Learn More MAT-0223 09/26

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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.

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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
Before taking AQNEURSA, tell your healthcare provider about all your medical conditions, including if you are pregnant or plan to become pregnant, or are breastfeeding or plan to breastfeed.
  • Based on findings from animal reproduction studies, AQNEURSA may cause harm to your unborn baby when administered during pregnancy.
  • Your healthcare provider will check to make sure you are not pregnant before starting treatment with AQNEURSA.
  • Use effective birth control (contraception) during treatment with AQNEURSA and for 7 days after stopping treatment to avoid pregnancy.
  • If you become pregnant while taking AQNEURSA, inform your healthcare provider immediately to discuss potential risks and alternative treatments.
  • It is not known if AQNEURSA or its metabolites pass into human or animal milk or effect breastmilk production.

Tell your healthcare provider about all the medications you take and if you get any new medications, including prescription and over-the-counter medicines, vitamins, and herbal supplements. Other medicines may cause side effects or affect how AQNEURSA works when used together.

  • Tell your healthcare provider if you are taking certain medications called P-gp substrates, N-acetyl-DL-leucine or N-acetyl-D-leucine. If you are not sure if you are taking these medicines, talk to your healthcare provider or pharmacist.

The most common side effects of AQNEURSA in people treated for Niemann-Pick disease type C include abdominal pain, dysphagia, upper respiratory tract infections and vomiting.

The most common side effects of AQNEURSA in people treated for ataxia-telangiectasia include fall, skin laceration, and urinary tract infection.

These are not all the possible side effects of AQNEURSA. Call your doctor for medical advice about side effects. You are encouraged to report side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch, or call 1-800-FDA-1088 .

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Important Safety Information
Before taking AQNEURSA, tell your healthcare provider about all your medical conditions, including if you are pregnant or plan to become pregnant, or are breastfeeding or plan to breastfeed.
  • Based on findings from animal reproduction studies, AQNEURSA may cause harm to your unborn baby when administered during pregnancy.
  • Your healthcare provider will check to make sure you are not pregnant before starting treatment with AQNEURSA.
  • Use effective birth control (contraception) during treatment with AQNEURSA and for 7 days after stopping treatment to avoid pregnancy.
  • If you become pregnant while taking AQNEURSA, inform your healthcare provider immediately to discuss potential risks and alternative treatments.
  • It is not known if AQNEURSA or its metabolites pass into human or animal milk or effect breastmilk production.

Tell your healthcare provider about all the medications you take and if you get any new medications, including prescription and over-the-counter medicines, vitamins, and herbal supplements. Other medicines may cause side effects or affect how AQNEURSA works when used together.

  • Tell your healthcare provider if you are taking certain medications called P-gp substrates, N-acetyl-DL-leucine or N-acetyl-D-leucine. If you are not sure if you are taking these medicines, talk to your healthcare provider or pharmacist.

The most common side effects of AQNEURSA in people treated for Niemann-Pick disease type C include abdominal pain, dysphagia, upper respiratory tract infections and vomiting.

The most common side effects of AQNEURSA in people treated for ataxia-telangiectasia include fall, skin laceration, and urinary tract infection.

These are not all the possible side effects of AQNEURSA. Call your doctor for medical advice about side effects. You are encouraged to report side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch, or call 1-800-FDA-1088 .