FDA Approves Revcovi, a New Enzyme Replacement Therapy Developed by Leadiant Biosciences, for the Treatment of ADA-SCID in Pediatric and Adult Patients

About Revcovi
Revcovi (elapegademase-lvlr) is a recombinant adenosine deaminase indicated for the treatment of adenosine deaminase severe combined immune deficiency (ADA-SCID) in pediatric and adult patients. In the two multicenter trials, Revcovi supplemented ADA levels, reduced concentrations of toxic metabolites that are the hallmark of ADA-SCID and improved total lymphocyte counts.1 Revcovi is the product of recombinant technology, thus eliminating the need to source the adenosine deaminase (ADA) enzyme from animals.Important Safety Information for Revcovi
Indication Revcovi (elapegademase-lvlr) is indicated for the treatment of adenosine deaminase severe combined immune deficiency (ADA-SCID) in pediatric and adult patients. WARNINGS AND PRECAUTIONS:- Injection site bleeding in patients with thrombocytopenia: Increased risk of local bleeding in patients with thrombocytopenia; should not be used if thrombocytopenia is severe.
- Delay in improvement of immune function: Protect immune deficient patients from infections until improvement in immune function.
- Hematologic events: hemolytic anemia, autoimmune hemolytic anemia, thrombocythemia, thrombocytopenia and autoimmune thrombocytopenia
- Dermatological events: injection site erythema, urticaria
- Lymphomas
About ADA-SCID
Adenosine deaminase severe combined immune deficiency (ADA-SCID) is an ultra-rare, inherited genetic disorder, caused by a deficiency in the adenosine deaminase (ADA) enzyme that is fatal when left untreated. ADA-SCID primarily affects infants and young children whose compromised immune system leaves them unprotected from infection-producing bacteria, viruses, and fungi. ADA-SCID is characterized by severe and recurrent opportunistic infections, failure to thrive, profound lymphopenia (reduced number of lymphocytes in the blood) with absent or severely impaired immune function, and metabolic abnormalities (abnormally high intracellular accumulation of purine nucleotides). Patients with ADA-SCID are also predisposed to recurrent illnesses caused by pathogens that often begin within the first few weeks of life.2,3ADA-SCID is typically diagnosed within the first few months of life.4 Left untreated, babies with ADA-SCID usually die before they reach the age of 2 due to recurrent infections unless they are diagnosed early and effective treatment is started.5 ADA-SCID results from mutations in the ADA gene, which provides instructions for producing the ADA enzyme.6 When functioning properly, ADA eliminates molecules called adenosine and deoxyadenosine, which are toxic to lymphocytes, a type of white blood cell. ADA converts adenosine to inosine and deoxyadenosine to deoxyinosine, molecules that do not harm lymphocytes. However, mutations in the ADA gene reduce or eliminate the protective activity of adenosine deaminase, allowing the buildup of adenosine and deoxyadenosine to toxic levels. These toxic levels cause specialized lymphocytes called T-cells and B-cells to accumulate biologic chemicals that would normally be processed by ADA. The buildup of these biologic products in excess of normal causes the T-cells and B-cells to die, leaving affected individuals with no significant immune defense and increasing their risk of infection.6 ADA-SCID is estimated to occur in approximately one in 200,000 to one in 1,000,000 newborns around the world.3 The disorder is responsible for approximately 15% of SCID cases.7About Leadiant Biosciences, Inc.
Leadiant Biosciences, Inc., a wholly-owned subsidiary of Leadiant Biosciences S.p.A., 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 and improve their quality of life. For additional information, please visit Leadiant.com.About Leadiant Biosciences, S.p.A. (Corporate)
Leadiant Biosciences, S.p.A. is a Rome-based global holding company with subsidiaries in the US (Leadiant Biosciences, Inc.) and Europe (Leadiant Biosciences, Ltd.) For additional information, please visit leadiantbiosciences.com. Contact at Leadiant Biosciences, Inc.: Nancy Parsons Leadiant Biosciences, Inc. Vice President, Pharmaceutical Products Phone: 301-670-2481 nancy.parsons@leadiant.com Media Alex Van Rees SmithSolve LLC on behalf of Leadiant Biosciences, Inc. Phone: 973-442-1555 ext. 111 alex.vanrees@smithsolve.com1 Revcovi? (elapegademase-lvlr) injection Prescribing Information. Leadiant Biosciences, Inc. 2018. 2 Booth C, Gaspar HB. Pegademase bovine (PEG-ADA) for the treatment of infants and children with severe combined immunodeficiency (SCID). Biologics Targets Ther. 2009;3:349-358. 3 Gaspar HB, Aiuti A, Porta F, Candotti F, Hershfield MS, Notarangelo LD. How I treat ADA deficiency. Blood. 2009;114:3524-3532. 4 Arredondo-Vega FX, Santisteban I, Daniels S, Toutain S, Hershfield MS. Adenosine deaminase deficiency: genotype-phenotype correlations based on expressed activity of 29 mutant alleles. Am J Hum Genet. 1998;63:1049-1059. 5 Hershfield MS. Adenosine deaminase deficiency. GeneReviews [Internet]. Initially posted October 2006. https://www.ncbi.nlm.nih.gov/books/NBK1483/. Accessed August 26, 2017. 6 Adenosine deaminase deficiency. Genetics Home Reference. Bethesda, MD: U.S. Department of Health and Human Services, National Institutes of Health, National Library of Medicine, Lister Hill National Center for Biomedical Communications; 2016. Available at: https://ghr.nlm.nih.gov/condition/adenosine-deaminase-deficiency. Accessed September 15, 2017. 7 Hershfield MS. Immunodeficiency caused by adenosine deaminase deficiency. Immunol Allergy Clin North Am. 2000;20:161-175.