Medications for Acid Sphingomyelinase Deficiency
Other names: ASMD; Sphingolipidosis
Acid sphingomyelinase deficiency (ASMD), historically known as Niemann-Pick disease types A, A/B, and B, is a lysosomal storage disease that results from reduced activity of the enzyme acid sphingomyelinase (ASM). It is an extremely rare, progressive genetic disease caused by pathogenic variants in the sphingomyelin phosphodiesterase 1 gene.
ASM is an enzyme that degrades sphingomyelin to ceramide and phosphocholine. The deficiency of ASM causes an intra-lysosomal accumulation of sphingomyelin (as well as cholesterol and other cell membrane lipids) in various tissues.
Signs and symptoms of ASMD can present in infancy, childhood, or adulthood, and may include enlarged spleen or liver, difficulty breathing, lung infections, and unusual bruising or bleeding, among other disease manifestations.
Xenpozyme (olipudase alfa-rpcp) is the first FDA-approved treatment for ASMD. It is a hydrolytic lysosomal sphingomyelin-specific enzyme replacement therapy, designed to replace deficient or defective acid sphingomyelinase (ASM).
Drugs used to treat Acid Sphingomyelinase Deficiency
The following list of medications are in some way related to or used in the treatment of this condition.
Frequently asked questions
Learn more about Acid Sphingomyelinase Deficiency
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|Activity is based on recent site visitor activity relative to other medications in the list.
|Prescription or Over-the-counter.
|This medication may not be approved by the FDA for the treatment of this condition.
|An Emergency Use Authorization (EUA) allows the FDA to authorize unapproved medical products or unapproved uses of approved medical products to be used in a declared public health emergency when there are no adequate, approved, and available alternatives.
|Expanded Access is a potential pathway for a patient with a serious or immediately life-threatening disease or condition to gain access to an investigational medical product (drug, biologic, or medical device) for treatment outside of clinical trials when no comparable or satisfactory alternative therapy options are available.
|Adequate and well-controlled studies have failed to demonstrate a risk to the fetus in the first trimester of pregnancy (and there is no evidence of risk in later trimesters).
|Animal reproduction studies have failed to demonstrate a risk to the fetus and there are no adequate and well-controlled studies in pregnant women.
|Animal reproduction studies have shown an adverse effect on the fetus and there are no adequate and well-controlled studies in humans, but potential benefits may warrant use in pregnant women despite potential risks.
|There is positive evidence of human fetal risk based on adverse reaction data from investigational or marketing experience or studies in humans, but potential benefits may warrant use in pregnant women despite potential risks.
|Studies in animals or humans have demonstrated fetal abnormalities and/or there is positive evidence of human fetal risk based on adverse reaction data from investigational or marketing experience, and the risks involved in use in pregnant women clearly outweigh potential benefits.
|FDA has not classified the drug.
|Controlled Substances Act (CSA) Schedule
|The drug has multiple schedules. The schedule may depend on the exact dosage form or strength of the medication.
|CSA Schedule is unknown.
|Is not subject to the Controlled Substances Act.
|Has a high potential for abuse. Has no currently accepted medical use in treatment in the United States. There is a lack of accepted safety for use under medical supervision.
|Has a high potential for abuse. Has a currently accepted medical use in treatment in the United States or a currently accepted medical use with severe restrictions. Abuse may lead to severe psychological or physical dependence.
|Has a potential for abuse less than those in schedules 1 and 2. Has a currently accepted medical use in treatment in the United States. Abuse may lead to moderate or low physical dependence or high psychological dependence.
|Has a low potential for abuse relative to those in schedule 3. It has a currently accepted medical use in treatment in the United States. Abuse may lead to limited physical dependence or psychological dependence relative to those in schedule 3.
|Has a low potential for abuse relative to those in schedule 4. Has a currently accepted medical use in treatment in the United States. Abuse may lead to limited physical dependence or psychological dependence relative to those in schedule 4.
|Interacts with Alcohol.
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