What are the precautions for levacetylleucine?

In the clinical research of levacetylleucine to treatNiemann-Pick disease type C (NPC; rare disease), special attention needs to be paid to embryo-fetal toxicity and other safety issues. Clinical studies have shown that this drug may cause embryo-fetal damage. Therefore, if related adverse reactions occur during use, it is necessary to consider suspending treatment and adjust the dose after the symptoms are relieved, or decide whether to permanently discontinue the drug based on the severity of the disease.

Animal reproduction studies have found that leacetylleucine may cause harm to the fetus when used during pregnancy. Administration of L-acetylleucine to pregnant rats and rabbits during the period of organogenesis increases embryonic-fetal mortality and causes skeletal malformations. Among them, pregnant rats experienced post-implantation loss and absorption after receiving a dose equivalent to 1.4 times the maximum recommended human dose (MRHD), while pregnant rabbits also experienced a similar situation after receiving a dose 6 times the MRHD. Therefore, the decision to continue leacetylleucine therapy during pregnancy needs to be weighed against the patient's need for the drug, the possible risks to the fetus, and the possible adverse consequences of untreated maternal disease.

For women of childbearing potential, it is necessary to confirm that they are not pregnant before starting treatment with levoacetylleucine. At the same time, patients are advised to take effective contraceptive measures during treatment to reduce fetal risks. If you stop using levoacetylleucine , you need to continue contraception within 7 days after the last dose to ensure that the drug will not affect the fetus. Taken together, althoughlevacetylleucinehas shown significant efficacy inNPC treatment, safety guidelines still need to be strictly followed during use, especially the management of women of childbearing age to ensure the safety of mothers and infants.

Reference materials:https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=0f248e55-d1bb-13f9-e063-6294a90a05ce