Safety

In the pivotal trial, AQNEURSA was well tolerated compared to placebo¹

There were no discontinuations due to adverse events in the robust clinical study program²

Most common adverse reactions (incidence ≥5%) with AQNEURSA in the pivotal trial¹

      
          Adverse reaction
          AQNEURSA
(n=30) n (%)
          Placebo
(n=30) n (%)
      
          Upper respiratory tract infection
          5 (17) 
          1 (3)
        
          Abdominal pain
          2 (7)
          0 (0)
        
          Dysphagia
          2 (7)
          0 (0)
        
          Vomiting
          2 (7)
          0 (0)

No serious adverse reactions were related to AQNEURSA²
During the trial, 1 patient experienced an exacerbation of rosacea that responded to treatment. AQNEURSA was not discontinued¹
Epilepsy, a feature of Niemann-Pick disease type C (NPC), occurred once when a patient was receiving AQNEURSA²
Thrombocytopenia (platelets <100 x 10³ cells/µL) was observed in 4 patients, all of whom had received miglustat for ≥42 days at the time of enrollment¹

Two patients had thrombocytopenia at baseline¹
Two patients developed thrombocytopenia during the trial¹

Explore dosing and administration

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

In Niemann-Pick disease type C, the most common adverse reactions (incidence ≥5% and greater than placebo) are abdominal pain, dysphagia, upper respiratory tract infections, and vomiting.
In ataxia-telangiectasia, the most common adverse reactions (incidence ≥5% and greater than placebo) are fall, skin laceration, and urinary tract infection.

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.

AQNEURSA^® (levacetylleucine) is indicated for the treatment of ataxia in adults and pediatric patients with ataxia-telangiectasia (A-T) 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