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Target intelligence / Profile preview
Acid Sphingomyelinase (ASM)
- Target
- ASM
- Molecular classification
- Enzyme, Sphingomyelinase, Hydrolase
01
Overview
Acid sphingomyelinase (ASM) is a lysosomal enzyme encoded by the SMPD1 gene. It catalyzes the hydrolysis of sphingomyelin into ceramide and phosphorylcholine. ASM plays crucial roles in lipid metabolism, cell signaling, and apoptosis. Deficiency in ASM leads to Niemann-Pick disease types A and B, characterized by sphingomyelin accumulation. Olipudase alfa is an enzyme replacement therapy approved for non-CNS manifestations. Functional inhibitors of ASM (FIASMAs) are being explored for conditions involving excessive ASM activity.
- Other names
- SMPD1Sphingomyelin phosphodiesterase 1L-SMaseS-SMase
02
Mechanism of action
Olipudase alfa: Enzyme replacement therapy; provides exogenous ASM to reduce sphingomyelin accumulation. FIASMAs: Inhibit ASM activity, reducing ceramide production.
03
Biological functions
Lipid metabolismSignal transductionApoptosisMembrane reorganizationImmune function modulationInflammation response
04
Disease associations
Acid Sphingomyelinase Deficiency (ASMD)Niemann-Pick disease types A and BNeurodegenerative diseaseLysosomal storage disorderDepressionInfectionImmune disorders
05
Safety considerations
Olipudase alfa: Infusion-related reactions, hypersensitivity.FIASMAs: Potential off-target effects due to broad inhibition of ceramide metabolism.Neurological involvement (limited CNS penetration of some therapies).
06
Interacting drugs
Olipudase alfa
1 more in the full profile.
07
Biomarkers
Sphingomyelin levels in tissues (e.g., liver, spleen)Chitotriosidase activity (elevated in ASMD)Lyso-Gb1 (elevated in ASMD)
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