Niemann-Pick type C (NPC) disease

Active Ingredient: Levacetylleucine

Indication for Levacetylleucine

Population group: only children (1 year - 12 years old) , adolescents (12 years - 18 years old) , adults (18 years old or older)
Therapeutic intent: Curative procedure

Levacetylleucine is indicated for the treatment of neurological manifestations of Niemann-Pick type C (NPC) disease, in combination with miglustat, or as a monotherapy in patients where miglustat is not tolerated, in adults and children aged 6 years and older and weighing at least 20 kg.

For this indication, competent medicine agencies globally authorize below treatments:

2-4 d daily in divided doses based on patient's body weight

For:

Dosage regimens

Regimen A, in case that patient age in years is ≥ 6 and patient weight is ≥ 20 kg and patient weight is ≤ 24 kg

Oral, 1 milligrams levacetylleucine, 2 times daily.

Regimen B, in case that patient age in years is ≥ 6 and patient weight is ≥ 25 kg and patient weight is ≤ 34 kg

Oral, 1 milligrams levacetylleucine, 3 times daily.

Regimen C, in case that patient age in years is ≥ 6 and patient weight is ≥ 35 kg

Oral, 4 milligrams levacetylleucine, divided 3 times daily.

Detailed description

Adults and paediatric population aged 6 years and older and weighing at least 20 kg

The recommended dose is based on the patient's body weight in kg according to the following table. Food and drink should be avoided 0.5 hours before and 2 hours after administration.

Recommended dose:

Patient's body weightMorning doseAfternoon doseEvening dose
20 to 24 kg1 gNo dose1 g
25 to 34 kg1 g1 g1 g
35 kg or more2 g1 g1 g

If a dose is missed, it should be skipped, and the next dose should be taken as scheduled.

Active ingredient

Levacetylleucine

Levacetylleucine is a modified amino acid that helps break down accumulated lipids in the brain (improves the function of lysosomes and mitochondria). Non-clinical studies demonstrated that levacetylleucine corrects energy metabolism, including improving the production of adenosine triphosphate. It is administered for the treatment of neurological manifestations of Niemann-Pick type C disease (NPC) in adults and children (≥15 kg).

Read more about Levacetylleucine

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