Aqneursa™
Drug - Aqneursa™ (levacetylleucine) [IntraBio Inc.]
June 2025
Therapeutic Area - Niemann-Pick disease type C (NPC)
Initial approval criteria
- Patient has a diagnosis of Niemann-Pick disease Type C; AND
- Genetic analysis confirms mutation in the NPC1 or NPC2 genes; AND
- Patient has disease-related neurological symptoms; AND
- Patient weighs greater than or equal to 15 kg AND
- Patient does not have any FDA labeled contraindications to Aqneursa; AND
- Prescriber is a specialist in the area of the patient’s diagnosis (e.g., geneticist, neurologist) or prescriber has consulted with a specialist in the area of patient’s diagnosis.
- Initial approval is for 6 months
Renewal criteria
- Patient has had clinical benefit with Aqneursa AND
- Patient does not have any FDA labeled contraindications to Aqneursa; AND
- Prescriber is a specialist in the area of the patient’s diagnosis (e.g., geneticist, neurologist) or prescriber has consulted with a specialist in the area of patient’s diagnosis
- Renewal approval is for 12 months
Quantity limits
- 1-gram levacetylleucine in a unit-dose packet: 136 packets per 34 days
- Patient’s weight (in kg) must be submitted at time of request
Questions?
Provider Call Center (844) 575-7887
