41. Revcovi


Active ingredient: elapegademase-lvlr 
Disease: adenosine deaminase severe combined immunodeficiency in pediatric and adult patients 
Peak sales estimate: n/a 
Approved: Oct. 5 
Company: Leadiant Biosciences 

The scoop: With Revcovi’s October FDA nod, Leadiant Biosciences added a second enzyme replacement therapy for adenosine deaminase severe combined immunodeficiency (ADA-SCID), a rare genetic disorder in which a lack of the enzyme adenosine deaminase (ADA) causes severe immunodeficiency. Its first treatment for the diseases—which is colloquially known as “bubble boy syndrome”—is Adagen, an enzyme replacement therapy approved in 1990 containing a bovine form of ADA that has been genetically modified to work in humans. Like Adagen, Revcovi works by replenishing adenosine deaminase, but it is a PEGylated recombinant adenosine deaminase (rADA) enzyme, which eliminates the need to use enzymes derived from animals. The approval was based on data from two open-label trials that showed Revcovi increased ADA activity, bolstered lymphocyte count and lowered concentrations of toxic metabolites that are the hallmark of ADA-SCID. — Amirah Al Idrus 

41. Revcovi

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