Logo for AQNEURSA™ (levacetylleucine), indicated for the treatment of adult and pediatric patients with neurological manifestations of NPC weighing 15 kg or moreLogo for AQNEURSA™ (levacetylleucine), indicated for the treatment of adult and pediatric patients with neurological manifestations of NPC weighing 15 kg or more

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AQNEURSA is the only FDA-approved stand-alone therapy for the treatment of NPC, demonstrating significant improvements in neurological symptoms and functional benefits that could be seen within 12 weeks in adult and pediatric patients1,2,*
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.

About Niemann-Pick disease type C (NPC)

  • NPC is a rare genetic disorder, occurring in about 1 in 100,000 live births3
  • NPC is defined by neurological, systemic, and psychiatric challenges that ultimately result in serious functional and cognitive decline3
  • AQNEURSA was studied in clinical trials to address the urgent need for a treatment that helps improve both the neurological and functional symptoms of NPC1-3
To learn more about NPC, explore these useful resources.

About AQNEURSA

AQNEURSA is a new treatment approved for adults and pediatric patients with NPC weighing 15 kg or more.¹
Efficacy
The efficacy of AQNEURSA was established in a pivotal study of 60 patients. Patients in the study received AQNEURSA for 12 weeks and placebo for 12 weeks. Some patients received the placebo first, while others received AQNEURSA first.1

In the pivotal study, AQNEURSA improved neurological symptoms and showed functional benefits that could be seen within 12 weeks when compared with responses to placebo.1,*

Safety
The safety of AQNEURSA was established in a robust clinical study program.1,2
The most common adverse reactions (incidence ≥5% and higher than placebo among the 30 patients in the first treatment period of the pivotal study) were abdominal pain (2 patients), dysphagia (difficulty swallowing, 2 patients), upper respiratory tract infections (5 patients), and vomiting (2 patients).1
Dosing and administration
AQNEURSA features oral dosing that fits into patients’ daily routines.1
To access AQNEURSA once prescribed, download and complete the Start AQNEURSA Prescription Form.
*Neurological symptoms and functional outcomes were assessed using a modified version of the Scale for Assessment and Rating of Ataxia (SARA), referred to as the functional SARA (fSARA). Patients who received AQNEURSA first in Period I followed by placebo second in Period II (Treatment Sequence 1) showed a greater improvement in the fSARA score in Period I, with a mean change from baseline of -0.5 (standard deviation: 1.2), compared to Period II with a mean change from baseline of 0 (1.5). Similarly, patients who received placebo first in Period I followed by AQNEURSA second in Period II (Treatment Sequence 2) experienced greater improvement in the fSARA score while receiving AQNEURSA in Period II, with a mean change of -0.7 (0.9), compared to a mean change of -0.3 (0.9) in Period I.1

Helpful resources

NPC organizations
National Niemann-Pick Disease Foundation
Ara Parseghian Medical Research Fund
International Niemann-Pick Disease Alliance
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.
Downloadable resources
Resources to help access an AQNEURSA prescription:
Resources with more information about AQNEURSA:
AQNEURSA Cares program

The AQNEURSA Cares program can help eligible patients access treatment.

Learn more by downloading the brochure that's right for you:

Logo for the AQNEURSA Cares program, which helps eligible patients access treatmentLogo for the AQNEURSA Cares program, which helps eligible patients access treatment
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.

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.

Please click here for Full Prescribing Information for AQNEURSA.

References:
  1. AQNEURSA. Prescribing information. IntraBio.
  2. Bremova-Ertl T, Ramaswami U, Brands M, et al. Trial of N-acetyl-L-leucine in Niemann-Pick disease type C. N Engl J Med. 2024;390(5):421-431. doi:10.1056/NEJMoa2310151
  3. Geberhiwot T, Moro A, Dardis A, et al; International Niemann-Pick Disease Registry (INPDR). Consensus clinical management guidelines for Niemann-Pick disease type C. Orphanet J Rare Dis. 2018;13(1):50. doi:10.1186/s13023-018-0785-7

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.

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-DLleucine 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.

Please click here for Full Prescribing Information for AQNEURSA.

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