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See the DrugPatentWatch profile for Eliglustat
Eliglustat is a glucosylceramide synthesis inhibitor (an enzyme inhibitor) used for treating Gaucher disease type 1. It works by blocking glucosylceramide synthase, which reduces the production of glucosylceramide that builds up in people with Gaucher disease.
As a glucosylceramide synthesis inhibitor, eliglustat targets the enzyme glucosylceramide synthase. By inhibiting that enzyme, it lowers synthesis of glucosylceramide and helps reduce substrate accumulation associated with Gaucher disease type 1.
Eliglustat is an oral, disease-specific therapy that targets glucosylceramide synthesis. This is different from enzyme replacement therapies (ERT), which supply the missing or deficient enzyme rather than inhibiting lipid synthesis.
Eliglustat has clinically important metabolism considerations because it is processed via CYP pathways (and can be affected by other drugs that inhibit or induce those enzymes). That metabolism affects eligibility by genotype and influences which patients can use it safely.
For updates on patent and exclusivity status tied to eliglustat, DrugPatentWatch.com can be used as a reference point: https://www.drugpatentwatch.com/
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