Access & resources

The AQNEURSA Cares program may help eligible patients access treatment

The AQNEURSA Cares team is ready to help assist your eligible patients with gaining access to AQNEURSA

Send a completed enrollment packet, including the Start AQNEURSA Prescription Form, to AQNEURSA Cares for the team to initiate the following services:

  • Verification of insurance coverage for AQNEURSA
  • Determination of eligibility for co-pay assistance
  • Support during the prior authorization process
  • Patient outreach and scheduling of medication shipments directly to the patient/caregiver

The complete enrollment packet consists of the Start AQNEURSA Prescription Form and the clinical information listed in the Prior Authorization Checklist.

Logo for the AQNEURSA™ Cares program, providing HCPs, patients and caregivers with patient support services
Fax the completed and signed Start AQNEURSA Prescription Form, along with the clinical information, to AQNEURSA Cares at 470-751-8639.
Questions? Call 1-866-200-0419 Monday–Friday, 8:30 am to 8 pm ET, for assistance in coordinating patient access to treatment.

Resources and support for your practice

Help your patients access AQNEURSA and get started on treatment.

Resources and support for your patients

Resources to help your patients set expectations for treatment with AQNEURSA and learn about support and services provided by IntraBio.

Patients, families, and caregivers can find additional information about NPC and connection with the community at the following NPC organizations:

IMPORTANT SAFETY INFORMATION
Embryo-Fetal Toxicity
  • Based on findings from animal reproduction studies, AQNEURSA may cause embryo-fetal harm when administered during pregnancy. The decision to continue or discontinue AQNEURSA treatment during pregnancy should consider the female’s need for AQNEURSA, the potential drug-related risks to the fetus, and the potential adverse outcomes from untreated maternal disease.
Pregnancy and Lactation
  • For females of reproductive potential, verify that the patient is not pregnant prior to initiating treatment with AQNEURSA. Advise females of reproductive potential to use effective contraception during treatment with AQNEURSA and for 7 days after the last dose if AQNEURSA is discontinued.
  • There are no data on the presence of levacetylleucine or its metabolites in either human or animal milk, the effects on the breastfed infant or the effects on milk production. The developmental and health benefits of breastfeeding should be considered along with the mother’s clinical need for AQNEURSA and any potential adverse effects on the breastfed infant from levacetylleucine or from the underlying maternal condition.
Adverse Reactions
  • The most common adverse reactions (incidence ≥5% and greater than placebo) are abdominal pain, dysphagia, upper respiratory tract infections, and vomiting.
Drug Interactions
  • Avoid concomitant use of AQNEURSA with N-acetyl-DL-leucine or N-acetyl-D-leucine. The D-enantiomer, N-acetyl-D-leucine, competes with levacetylleucine for monocarboxylate transporter uptake, which may reduce the levacetylleucine efficacy.
  • Monitor more frequently for P-gp substrate related adverse reactions when used concomitantly with AQNEURSA; AQNEURSA inhibits P-gp; however, the clinical significance of this finding has not been fully characterized.
To report SUSPECTED ADVERSE REACTIONS, contact IntraBio Inc. at 1-833-306-9677 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.
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.

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. MIPLYFFA. Prescribing information. Zevra Therapeutics Inc; 2024. 4. 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 5. Burton BK, Ellis AG, Orr B, et al. Estimating the prevalence of Niemann-Pick disease type C (NPC) in the United States. Mol Genet Metab. 2021;134:182-187. doi:10.1016/j.ymgme.2021.06.011 6. Kassen S, Parseghian C, Andrews P, et al. Niemann-Pick Type C Patient and Caregiver Voices: Externally-led, Patient-focused Drug Development Meeting. The Ara Parseghian Medical Research Fund at Notre Dame; 2019.

Back to top

Important Safety Information

Embryo-Fetal Toxicity
  • Based on findings from animal reproduction studies, AQNEURSA may cause embryo-fetal harm when administered during pregnancy. The decision to continue or discontinue AQNEURSA treatment during pregnancy should consider the female’s need for AQNEURSA, the potential drug-related risks to the fetus, and the potential adverse outcomes from untreated maternal disease.
Pregnancy and Lactation
  • For females of reproductive potential, verify that the patient is not pregnant prior to initiating treatment with AQNEURSA. Advise females of reproductive potential to use effective contraception during treatment with AQNEURSA and for 7 days after the last dose if AQNEURSA is discontinued.
  • There are no data on the presence of levacetylleucine or its metabolites in either human or animal milk, the effects on the breastfed infant or the effects on milk production. The developmental and health benefits of breastfeeding should be considered along with the mother’s clinical need for AQNEURSA and any potential adverse effects on the breastfed infant from levacetylleucine or from the underlying maternal condition.
Adverse Reactions
  • The most common adverse reactions (incidence ≥5% and greater than placebo) are abdominal pain, dysphagia, upper respiratory tract infections, and vomiting.
Drug Interactions
  • Avoid concomitant use of AQNEURSA with N-acetyl-DL-leucine or N-acetyl-D-leucine. The D-enantiomer, N-acetyl-D-leucine, competes with levacetylleucine for monocarboxylate transporter uptake, which may reduce the levacetylleucine efficacy.
  • Monitor more frequently for P-gp substrate related adverse reactions when used concomitantly with AQNEURSA; AQNEURSA inhibits P-gp; however, the clinical significance of this finding has not been fully characterized.
To report SUSPECTED ADVERSE REACTIONS, contact IntraBio Inc. at 1-833-306-9677 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

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.
Logo for IntraBio, a biopharmaceutical company focused on therapies for neurodegenerative diseases with high unmet medical needs

This site is intended for US healthcare professionals only.

AQNEURSA, AQNEURSA Cares, and their respective logos are trademarks of IntraBio.

© 2026 IntraBio. All rights reserved. MAT-0052 04/26

© 2025 IntraBio. All rights reserved. MAT-0052 01/25

Austin, TX

Contact Us
Privacy Policy
Cookie Policy