Support and resources

AQNEURSA Cares program

The AQNEURSA Cares program can help eligible patients access treatment. It is the single point of contact for access to AQNEURSA, and the team is there to help you or someone you care for get started once prescribed.

Logo for the AQNEURSA™ Cares program, providing patients and caregivers with patient support services

Get to know the program by downloading the brochure, and work with the prescribing healthcare provider to complete the starter form.

Helpful resources

Review these useful resources that can help with understanding and getting started on AQNEURSA.

Niemann-Pick disease type C (NPC) organizations

Learn more about the disorder and connect with the NPC community.

IntraBio is not responsible for the content or services of these organizations. Inclusion of these links on this website is not intended as an endorsement of the organizations or the services offered.

Helpful tip

The NPC community is not just there for people with NPC, it’s also there for families and those who provide care.

See answers to frequently asked questions

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 .

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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.