Leadiant Biosciences, Inc. today announced that the Food and Drug Administration (FDA) has granted approval to Revcovi™ (elapegademase-lvlr) injection in the U.S. Revcovi is a new enzyme replacement therapy (ERT) 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. "We extend our deepest gratitude to the patients who participated in the clinical trials and their families and caregivers who supported them. We also appreciate the hard work of the investigators, clinicians, and study staff to bring this therapy to patients in need. We look forward to continuing to work together to serve the ADA-SCID community."
Revcovi is a PEGylated recombinant adenosine deaminase (rADA) 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).
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.
"For decades, physicians, patients, and their families have relied upon enzyme replacement therapy as a life-saving treatment for adenosine deaminase severe combined immunodeficiency, a disease in which the buildup of toxic metabolites can cripple children's immune systems," said Morna Dorsey, M.D., MMSc, Professor of Pediatrics at the University of California, San Francisco. "Individuals with ADA-SCID are at an increased risk of severe and recurrent infections and often fail to thrive. By providing specific and direct replacement of the adenosine deaminase enzyme, Revcovi can reduce patients' risk of potentially serious, life-threatening infections and their debilitating complications."
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.
"This is a great day for people living with ADA-SCID and their families as the approval of Revcovi gives them a path forward," commented John Boyle, President and Chief Executive Officer of the Immune Deficiency Foundation. "We commend Leadiant Biosciences for bringing this innovative enzyme replacement therapy to market, and for helping to advance scientific understanding of ADA-SCID."
"The competence and dedication of our staff was instrumental to obtain this important achievement for the ADA-SCID community," said Dr. Marco Brughera, Chief Executive Officer, Leadiant Biosciences Corporate. "With the FDA's approval of Revcovi, we reaffirm our commitment and rare dedication to providing a reliable supply of quality, innovative therapies that serve the needs of rare disease communities."
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. 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.