Frequently asked questions

Answers to some common questions about AQNEURSA

These answers are not intended to replace a discussion about AQNEURSA with a healthcare provider.

AQNEURSA may be appropriate for adults and children with Niemann-Pick disease type C (NPC) who weigh 15 kg (33 lb) or more to treat the neurological manifestations of the disorder. If you are pregnant or planning to become pregnant, or are breastfeeding or planning to breastfeed, talk to your doctor before starting AQNEURSA.

As symptoms of NPC vary, the results with AQNEURSA, if any, will differ depending on the person. In the clinical trial of 60 people with NPC, AQNEURSA improved many activities of daily living that support everyday functional independence, including walking, balance, speech, and fine motor skills, within 12 weeks compared with placebo.

In the clinical trial, people with NPC who were given AQNEURSA were evaluated after 6 weeks, and again after 12 weeks, of treatment. 
The most common side effects of AQNEURSA include abdominal pain (pain in the belly or stomach area), dysphagia (trouble swallowing), upper respiratory tract infections (infections of the nose, mouth, or throat), and vomiting. Speak with your healthcare provider if you or someone you care for experiences these side effects and if these side effects persist or worsen.
AQNEURSA is taken by mouth or via a gastrostomy tube (G-tube) 2 to 3  times per day. The drug should be taken exactly as prescribed. AQNEURSA can be taken with or without food.
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 the missed dose.

The AQNEURSA Cares team is there to help eligible patients determine if AQNEURSA is covered by their health insurance. To get started, you or someone you care for must review and complete the Start AQNEURSA Prescription Form with the prescribing healthcare provider, who will submit the form. The AQNEURSA Cares team will verify your insurance coverage, determine if you are eligible for co-pay assistance, and let you know your options.

To enroll in AQNEURSA Cares, you or someone you care for must review and complete the Start AQNEURSA Prescription Form with the prescribing healthcare provider, who will submit the form. A representative from the AQNEURSA Cares team will contact you to help with next steps.

“There were significant improvements in our daily life. Regular activities are possible more often.”

– Parents of a child treated with AQNEURSA in the clinical trial. This is an individual's experience and cannot be considered to reflect the entire population.

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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 .

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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.