Research & Development
FDA Approves Revcovi, a New Enzyme Replacement Therapy for the Treatment of ADA-SCID in Pediatric and Adult Patients
9 October 2018 - - The Food and Drug Administration has granted approval to Revcovi (elapegademase-lvlr) injection in the US, its developer, US-based Leadiant Biosciences, Inc., said.

Revcovi is a new enzyme replacement therapy for the treatment of adenosine deaminase severe combined immune deficiency (ADA-SCID) in pediatric and adult patients.

Revcovi is a PEGylated recombinant adenosine deaminase enzyme developed by Leadiant Biosciences to treat ADA-SCID.

The product of recombinant technology, Revcovi eliminates the need to source the enzyme from animals and works by supplementing levels of an essential enzyme called adenosine deaminase.

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.

SCID newborn screening in most states has allowed detection of ADA-SCID in newborns and has led to early initiation of ADA enzyme therapy and improved outcomes.

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 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.

This initiative will help Leadiant better understand and track information about Revcovi following approval as well as provide critical information about Revcovi's efficacy and safety, especially in newly diagnosed patients.

Leadiant is a research-based pharmaceutical company that dedicates considerable scientific and financial resources to the research, development, and distribution of novel and effective therapies to address the needs of people living with rare diseases.

The company markets five rare disease products in North America and has been working in the enzyme replacement therapy space for more than 30 years. The company is committed to serving the needs of patients, caregivers, and families affected by ADA-SCID.

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