Elapegademase for ADA-SCIDDecember 13, 2018
US FDA has granted approval for elapegademase (Revcovi) for adenosine deaminase severe combined immune deficiency (ADA-SCID).
Elapegademase is a long-acting drug that replaces the missing ADA enzyme in patients with ADA-SCID with an engineered, recombinant form.
The new drug comes as an alternative to current animal-derived enzyme replacement therapy pegademase bovine by Leadiant.
Also known as bubble boy disease, ADA-SCID is an ultra-rare, inherited geneticdisorder that compromises patients’ immune systems and leaves patients unprotected from infections, and is fatal if untreated.
The disease is typically diagnosed within the first few months of life, and undiagnosed babies with ADA-SCID usually die before they reach age two due to infections.
Elapegademase approval is based on two open-label trials demonstrating the drug increases ADA activity, reduces concentrations of toxic metabolites and improves total lymphocyte counts in ADA-SCID patients.