About AQNEURSA

AQNEURSA is a first-in-class, chemically modified amino acid approved for use in Niemann-Pick disease type C (NPC)^1-3

The distinct molecular target for AQNEURSA in the treatment of NPC is unknown¹

The proposed mechanism of action (MOA) for AQNEURSA is the activation of cerebral glucose metabolism in the cerebellum, correlated with enhanced cerebellar activity^4,5
AQNEURSA is designed to enter enzyme-controlled pathways to correct metabolic dysfunction, improve lysosomal function, and enhance mitochondrial function and adenosine triphosphate (ATP) production^5,6

Desktop illustrative depiction of AQNEURSA's mechanism of action in preclinical studies

Mobile illustrative depiction of AQNEURSA's mechanism of action in preclinical studies.

Explore clinical data

ATP, adenosine triphosphate.

References: 1. AQNEURSA. Prescribing information. IntraBio. 2. Bremova-Ertl T, Rohrbach M, Ramaswami U, et al. Long-term findings of N-acetyl-L-leucine for Niemann-Pick disease type C. Presented at: 10th Congress of the European Academy of Neurology; June 29-July 2, 2024; Helsinki, Finland. 3. MIPLYFFA. Prescribing information. Zevra Therapeutics Inc; 2024. 4. Fields T, Patterson M, Bremova-Ertl T, et al. A master protocol to investigate a novel therapy acetyl-L-leucine for three ultra-rare neurodegenerative diseases: Niemann-Pick type C, the GM2 gangliosidoses, and ataxia telangiectasia. Trials. 2021;22(1):84. doi:10.1186/s13063-020-05009-3 5. Kaya E, Smith DA, Smith C, et al. Acetyl-leucine slows disease progression in lysosomal storage disorders. Brain Commun. 2020;3(1):fcaa148. doi:10.1093/braincomms/fcaa148 6. 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 7. Hegdekar N, Lipinski MM, Sarkar C. N-acetyl-L-leucine improves functional recovery and attenuates cortical cell death and neuroinflammation after traumatic brain injury in mice. Sci Rep. 2021;11(1):9249. doi:10.1038/s41598-021-88693-8 8. Newton J, Milstien S, Spiegel S. Niemann-Pick type C disease: the atypical sphingolipidosis. Adv Biol Regul. 2018;70:82-88. doi:10.1016/j.jbior.2018.08.001

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.

Important Safety Information