How to take AQNEURSA

AQNEURSA is an oral medication that can fit into your daily routine

  • AQNEURSA is taken as an oral suspension, which means the drug is dissolved in a liquid before drinking it
  • How much and how often AQNEURSA is taken depends on the body weight of the person prescribed the drug
Icon showing how AQNEURSA fits into a daily routine

Important information to know before taking AQNEURSA

Before taking AQNEURSA for the first time, and every time the prescription is refilled, read the Instructions for Use.
Icon showing how to take AQNEURSA

Take or give AQNEURSA exactly as instructed by your healthcare provider

Icon indicating that AQNEURSA can be taken with or without food

Take AQNEURSA with or without food

Icon representing intake with AQNEURSA by mouth or via a gastrostomy tube (G-tube)

Take AQNEURSA by mouth or via a gastrostomy tube (G-tube)

Icon indicating not to dissolve AQNEURSA in hot liquid

Do not dissolve AQNEURSA in hot liquid

How much AQNEURSA to take and how often to take it

The amount of AQNEURSA taken and how many times it is taken each day is based on body weight.

If the person weighs 15 kg (33 lb) or more,
but less than 25 kg (55 lb):		 If the person weighs 25 kg (55 lb) or more, but less than 35 kg (77 lb): If the person weighs 35 kg (77 lb) or more:
2 times a day:
1 packet every morning,
1 packet every evening 		3 times a day:
1 packet every morning,
1 packet every afternoon,
1 packet every evening 		3 times a day:
2 packets every morning,
1 packet every afternoon,
1 packet every evening

One packet contains 1 gram of AQNEURSA. If 2 packets of AQNEURSA are needed, prepare and take each packet individually, one after the other.

AQNEURSA should be taken around the same times each day, as prescribed. If a dose of AQNEURSA is missed, that missed dose should be skipped and the next dose taken as normal.

Do not take 2 doses at the same time to make up for a missed dose.

AQNEURSA for oral suspension is supplied in packets that are stored at room temperature, 20 °C to 25 °C (68 °F to 77 °F).

Helpful tip

If you care for someone with NPC, prepare each dose of AQNEURSA for them and supervise them as they take the mixture. It's important that they take all of the mixture immediately after you have prepared it (within 30 minutes).

Overview of how to prepare and take AQNEURSA

See the Instructions for Use for full details. Talk to your healthcare provider if you have additional questions about how to prepare and take AQNEURSA.

Preparing AQNEURSA

  • If taken by mouth: Empty the entire contents of 1 AQNEURSA packet into a container with 40 mL of water, orange juice, or almond milk, then stir
  • If given by G-tube: Empty the entire contents of 1 AQNEURSA packet into a container with 40 mL of water, then stir—do not dissolve in any liquid except for water
Illustration of stirring a drink, part of dosing instructions for AQNEURSA

Taking AQNEURSA

  • If taken by mouth: Drink all of the liquid containing AQNEURSA right away, within 30 minutes
Illustration of the correct method to drink AQNEURSA after preparation
  • If given by G-tube: Take up all of the liquid containing AQNEURSA into a syringe and inject into the G-tube right away, then flush the G-tube with 20 mL of water using the same syringe
Illustration of AQNEURSA dosing via G-tube

Throw away any remaining oral suspension (liquid mixture containing AQNEURSA) if not taken or given within 30 minutes.

Helpful tip

If you care for someone with NPC who has been prescribed AQNEURSA, it’s important that you make sure they take the right amount (number of packets) at the right time, as directed by their healthcare provider. AQNEURSA may be taken with or without food.

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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 .