Rare Disease Report

FDA Approves Elapegademase for Treatment of ADA-SCID in Pediatric and Adult Patients

OCTOBER 05, 2018
Krista Rossi
The FDA has approved Leadiant Biosciences, Inc. elapegademase-lvlr injection (Revcovi) for the treatment of adenosine deaminase severe combined immune deficiency (ADA-SCID) in pediatric and adult patients.

“We are gratified by the FDA’s timely recognition of Revcovi as an effective and safe treatment for ADA-SCID, which, in addition to being ultra-rare, is one of the most devastating genetic disorders,” said Michael Minarich, Chief Executive Officer, Leadiant Biosciences, Inc., in a recent statement.
 
Two multicenter, open-label clinical trials served as the basis for the approval. The trials demonstrated that the elapegademase-lvlr injection increases ADA activity, reduces concentrations of toxic metabolites that are the hallmark of ADA-SCID, and improves total lymphocyte counts.

A PEGylated recombinant adenosine deaminase (rADA) enzyme, the elapegademase-lvlr injection is engineered to treat ADA-SCID. Elapegademase-lvlr functions by supplementing levels of an essential enzyme called adenosine deaminase (ADA), eliminating the need to source the enzyme from animals as a result.

ADA-SCID is an inherited genetic disorder that is ultra-rare. It Is characterized by a deficiency in the ADA enzyme, which can lead to morbidity if left untreated.

Patients with ADA-SCID are often unprotected from infection-producing bacteria, viruses, and fungi since they have compromised immune systems. ADA-SCID is typically diagnosed within the first few months of life; undiagnosed infants often die of infections before the age of 2.

The detection of ADA-SCID in newborns has been provided by SCID newborn screening in most states and has led to early initiation of ADA enzyme therapy and improved outcomes as a result.

“Individuals with ADA-SCID are at an increased risk of severe and recurrent infections and often fail to thrive,” added Morna Dorsey, M.D., MMSc, Professor of Pediatrics at the University of California, San Francisco. “By providing specific and direct replacement of the adenosine deaminase enzyme, elapegademase-lvlr can reduce patients’ risk of potentially serious, life-threatening infections and their debilitating complications.”

Previously, the elapegademase-lvlr injection was granted a fast track. Priority review, and orphan drug designation.

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