FDA Approves Leadiant Biosciences’ Enzyme Replacement Therapy for ADA-SCID


Rare Daily Staff

The U.S. Food and Drug Administration approved Revcovi, Leadiant Biosciences’ enzyme replacement therapy for the treatment of adenosine deaminase severe combined immune deficiency (ADA-SCID) in pediatric and adult patients.

ADA-SCID is an ultra-rare, inherited genetic disorder, caused by a deficiency in the ADA enzyme that is fatal if left untreated. Patients affected by ADA-SCID have compromised immune systems that leave them unprotected from infection-producing bacteria, viruses, and fungi. ADA-SCID primarily affects infants and young children. The disease is typically diagnosed within the first few months of life. Undiagnosed babies with ADA-SCID usually die before they reach age two due to infections.

Revcovi is a PEGylated recombinant adenosine deaminase enzyme developed by Leadiant Biosciences to treat ADA-SCID. Revcovi eliminates the need to source the enzyme from animals and works by supplementing levels of an essential enzyme deficient in people with the condition.

“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, CEO of Leadiant Biosciences.

The approval is based on results from two multicenter, open-label clinical trials which demonstrate that Revcovi increases ADA activity, reduces concentrations of toxic metabolites that are the hallmark of ADA-SCID and improves total lymphocyte counts.

Leadiant is working with physicians, payers, and policymakers to bring Revcovi to patients who need it. The company offers comprehensive treatment support, from educating about the disease, to navigating reimbursement, to offering patient assistance programs. The company’s post-marketing commitment includes a clinical study, which will record information about the health status of patients using Revcovi.

The FDA granted this application Fast Track and Priority Review. Revcovi also received Orphan Drug designation.

October 8, 2018
Photo: Michael Minarich, CEO of Leadiant Biosciences

Filed Under: Business, Drug Development, Innovation

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