Medications for Niemann-Pick Disease
These are drugs that have been approved by the US Food and Drug Administration (FDA), meaning they have been determined to be safe and effective for use in Niemann-Pick Disease.
Found 3 Approved Drugs for Niemann-Pick Disease
Miplyffa
Generic Name
Arimoclomol
Miplyffa
Generic Name
Arimoclomol
Form: Capsule
Method of administration: Oral
FDA approval date: September 20, 2024
MIPLYFFA is indicated for use in combination with miglustat for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in adult and pediatric patients 2 years of age and older. MIPLYFFA is indicated for use in combination with miglustat for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in adult and pediatric patients 2 years of age and older. ( 1 )
Xenpozyme
Generic Name
Olipudase Alfa-Rpcp
Xenpozyme
Generic Name
Olipudase Alfa-Rpcp
Form: Injection
Method of administration: Intravenous
FDA approval date: August 29, 2022
XENPOZYME is indicated for treatment of non–central nervous system manifestations of acid sphingomyelinase deficiency (ASMD) in adult and pediatric patients. XENPOZYME is a hydrolytic lysosomal sphingomyelin-specific enzyme indicated for treatment of non–central nervous system manifestations of acid sphingomyelinase deficiency (ASMD) in adult and pediatric patients. ( 1 )
Aqneursa
Generic Name
Levacetylleucine
Aqneursa
Generic Name
Levacetylleucine
Form: Granule
Method of administration: Oral
FDA approval date: September 24, 2024
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 )
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