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
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 .