AQNEURSA

2 DOSAGE AND ADMINISTRATION • For females of reproductive potential, verify that the patient is not pregnant prior to initiating treatment. 2 ) Patient Body Weight Morning Dose Afternoon Dose Evening Dose 15 to <25 kg 1 g No Dose 1 g 25 to <35 kg 1 g 1 g 1 g 35 kg or more 2 g 1 g 1 g • See the full prescribing information for administration instructions.

3 )]. 2 Recommended Dosage The recommended dosage of AQNEURSA is based on the patient’s actual body weight (kg) to be administered orally up to three times daily. See Table 1 . AQNEURSA can be taken with or without food [see Clinical Studies ( 14 )] .

3 )] . Table 1 Recommended Dosage of Levacetylleucine Based on Body Weight (kg) Patient’s Body Weight Morning Dose Afternoon Dose Evening Dose 15 kg to less than 25 kg 1 gram No Dose 1 gram 25 kg to less than 35 kg 1 gram 1 gram 1 gram 35 kg or more 2 gram 1 gram 1 gram One AQNEURSA packet contains 1 gram levacetylleucine.

Missed Dose 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. 3 Preparation and Administration Instructions Oral Administration For oral administration, administer AQNEURSA as follows: 1.

Obtain the required number of AQNEURSA packets for the prescribed dose (one or two packets). 2. Open and empty the entire contents of one AQNEURSA packet into a container with 40 mL of water, orange juice, or almond milk. Do not use hot liquid.

3. Stir to form a suspension. 4. Swallow the suspension immediately (within 30 minutes). 5. For doses requiring two AQNEURSA packets, repeat steps 2 to 4. 6. Discard unused AQNEURSA suspension if not administered within 30 minutes. Use of Gastrostomy Tube (G-Tube) for Feeding Tube Administration For patients who have a G-tube (French size 18 or larger) in place, administer AQNEURSA as follows: 1.

Prepare AQNEURSA suspension immediately before administration via gastrostomy tube. 2. Obtain the required number of AQNEURSA packets for the prescribed dose (one or two packets). 3. Open and empty the entire contents of one AQNEURSA packet into a container with 40 mL of water ONLY.

Do not use hot liquid. 4. Stir to form a suspension. 5. Draw up the suspension into a catheter tip syringe. 6. Administer the suspension immediately through the G-tube. 7. Flush any residual suspension in the catheter tip syringe with an additional 20 mL of water.

6 ADVERSE REACTIONS Most common adverse reactions (incidence ≥5% and greater than placebo) are abdominal pain, dysphagia, upper respiratory tract infections, and vomiting. ( 6 ) To report SUSPECTED ADVERSE REACTIONS, contact IntraBio Inc.

gov/medwatch . 1 Clinical Trials Experience Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in the clinical trials of a drug cannot be directly compared to rates in the clinical trials of another drug and may not reflect the rates observed in practice.

The safety of AQNEURSA was evaluated in Trial 1, which included a total of 60 patients with Niemann-Pick disease Type C (NPC), in a placebo-controlled, randomized, crossover trial [see Clinical Studies ( 14 )] . 8) days (78 min, 113 max).

Table 2 summarizes adverse reactions that occurred in patients who were treated with AQNEURSA in Treatment Period I of Trial 1. Table 2 Adverse Reactions that Occurred in Adult and Pediatric Patients with NPC at an Incidence of ≥5% in Treatment Period I of Trial 1 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) Rosacea One patient experienced an exacerbation of rosacea during Trial 1 that responded to treatment.

AQNEURSA was not discontinued. Laboratory Findings Thrombocytopenia with platelets < 100 10^3 cells/µL was observed in four patients during Treatment Period 1, all of whom were receiving miglustat for 42 days or longer at the time of enrollment.

In two of these patients, the thrombocytopenia was present at baseline. In the other two patients, the thrombocytopenia developed during the trial.

5 WARNINGS AND PRECAUTIONS Embryo-Fetal Toxicity : May cause fetal harm. Advise females of reproductive potential to use effective contraception during treatment and for 7 days after the last dose if AQNEURSA is discontinued. 1 Embryo-Fetal Toxicity Based on findings from animal reproduction studies, AQNEURSA may cause embryo-fetal harm when administered during pregnancy.

4-fold and 6-fold, respectively, the maximum recommended human dose (MRHD) of 4 g/day of levacetylleucine (based on body surface area). 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.

For females of reproductive potential, verify that the patient is not pregnant prior to initiating treatment with AQNEURSA. 3 )].

4 CONTRAINDICATIONS None. None. ( 4 )