Dosing

AQNEURSA features oral dosing and administration that fit into patients’ daily routines¹

Weight-based dosing enables use across the broad age range of patients with Niemann-Pick disease type C (NPC)¹

Recommended dosage and schedule of AQNEURSA based on body weight¹

  
          Body weight
          Morning dose
          Afternoon dose
          Evening dose
        
          15 kg to <25 kg 
          1 gram
          No dose 
          1 gram 
        
          25 kg to <35 kg 
          1 gram
          1 gram
          1 gram
          
          ≥35 kg
          2 grams
          1 gram
          1 gram

AQNEURSA is administered by fully suspending a dose into 1 of a variety of cold or room-temperature liquids (water, orange juice, or almond milk)¹
AQNEURSA can be taken with or without food¹
Each AQNEURSA packet contains 1 gram of levacetylleucine¹
For 2-gram doses in patients ≥35 kg, 2 packets of AQNEURSA should be prepared and taken individually¹
If a dose is missed, the missed dose should be disregarded and the next dose should be taken as scheduled—patients should not take 2 doses at the same time to make up for a missed dose¹

In addition to oral administration, AQNEURSA may be administered via a gastrostomy tube (G-tube)¹

Review oral suspension directions

Oral administration¹

For oral administration, administer AQNEURSA as follows:

Obtain the required number of AQNEURSA packets for the prescribed dose (1 or 2 packets).
Open and empty the entire contents of 1 AQNEURSA packet into a container with 40 mL of water, orange juice, or almond milk. Do not use hot liquid.
Stir to form a suspension.
Swallow the suspension immediately (within 30 minutes).
For doses requiring 2 AQNEURSA packets, repeat steps 2 through 4.
Discard unused AQNEURSA suspension if not administered within 30 minutes.

Review G-tube directions

G-tube administration¹

For patients who have a G-tube (French size 18 or larger) in place, administer AQNEURSA as follows:

Prepare AQNEURSA suspension immediately before administration via G-tube.
Obtain the required number of AQNEURSA packets for the prescribed dose (1 or 2 packets).
Open and empty the entire contents of 1 AQNEURSA packet into a container with 40 mL of water ONLY. Do not use hot liquid.
Stir to form a suspension.
Draw up the suspension into a catheter-tip syringe.
Administer the suspension immediately through the G-tube.
Flush any residual suspension in the catheter-tip syringe with an additional 20 mL of water.
Flush the G-tube again, as needed, until no residual suspension is left in the syringe or feeding tube.
For doses requiring 2 AQNEURSA packets, repeat steps 3 through 8.
Discard unused AQNEURSA suspension if not administered immediately.

View full product information for AQNEURSA in the Instructions for Use and Full Prescribing Information.

Access helpful resources for you and your patients

Reference: 1. AQNEURSA. Prescribing information. IntraBio.

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