Aqneursa

AQNEURSA™ is indicated for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in adults and pediatric patients weighing ≥15 kg. AQNEURSA is indicated for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in adults and pediatric patients weighing ≥15 kg. ( 1 )

None. None. ( 4 )

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. ( 5.1 )

5.1Embryo-Fetal Toxicity Based

on findings from animal reproduction studies, AQNEURSA may cause embryo-fetal harm when administered during pregnancy. Administration of levacetylleucine to pregnant rats and rabbits during the period of organogenesis caused an increase in embryo-fetal death (post implantation loss/resorption) and skeletal malformations at a dose that was approximately 1.4-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. 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 [see Use in Specific Populations ( 8.1 , 8.3 )].

• N-acetyl-DL-leucine or N-acetyl-D-leucine : Avoid concomitant use with AQNEURSA. ( 7.1 )
• P-glycoprotein (P-gp) Transporter Substrates : Monitor for adverse reactions if used with AQNEURSA. ( 7.2 )

7.1Effect of Other Drugs on