Drug class

Metabolic Disease Enzyme Replacement, Severe Comb Immune Def

The phrase "Metabolic Disease Enzyme Replacement, Severe Comb Immune Def" is a specialized formulary and compendia classification rather than a standard pharmacological drug class designation. Read more about Metabolic Disease Enzyme Replacement, Severe Comb Immune Def

1 drugs in this class 1 manufacturers

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Drugs in Metabolic Disease Enzyme Replacement, Severe Comb Immune Def

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REVCOVI (elapegademase-lvlr)

METABOLIC DISEASE ENZYME REPLACEMENT, SEVERE COMB IMMUNE DEF · ADENOSINE DEAMINASE SEVERE COMBINED IMMUNE DEFICIENCY
CHIESI USA, INC
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About Metabolic Disease Enzyme Replacement, Severe Comb Immune Def.

The phrase "Metabolic Disease Enzyme Replacement, Severe Comb Immune Def" is a specialized formulary and compendia classification rather than a standard pharmacological drug class designation.

It encompasses enzyme replacement therapies, such as pegademase bovine and recombinant elapegademase, indicated specifically for adenosine deaminase-deficient severe combined immunodeficiency (ADA-SCID).

These agents function as exogenous substitutes for deficient adenosine deaminase, catabolizing toxic purine metabolites like adenosine and deoxyadenosine to alleviate lymphotoxicity and help reconstitute cellular and humoral immune function.

Clinically, these therapies are primarily utilized to stabilize metabolic and immunological status in infants and children affected by ADA-SCID, often serving as a bridge to definitive treatment options such as hematopoietic stem cell transplantation or gene therapy.