Breaking News
October 20, 2018 - Leading hip-hop videos depict use of tobacco and marijuana products, study finds
October 20, 2018 - Dose Range of IV Ketamine for Adjunct Tx of Depression Tested
October 20, 2018 - Infants can distinguish between leaders and bullies, study finds
October 20, 2018 - Mad Cow disease found on Aberdeenshire farm
October 20, 2018 - Study identifies factors associated with prescription opioid misuse among students
October 20, 2018 - Scientists uncover key regulator of mTORC1 in cancer growth
October 20, 2018 - Pounds Regained After Weight-Loss Op Can Tell Your Doc a Lot
October 20, 2018 - Sending parents letters to fight childhood obesity doesn’t work
October 20, 2018 - Supervised aerobic exercise can support major depression treatment
October 20, 2018 - Mindfulness-based program effective for reducing stress in infertile women
October 20, 2018 - Molecule capable of halting and reverting neurodegeneration caused by Parkinson’s disease identified
October 20, 2018 - Midazolam-mediated alterations of PER2 expression may have functional consequences during myocardial ischemia
October 20, 2018 - Sweat bees are ideal for studying the genes underlying social behavior
October 20, 2018 - Weight loss success associated with brain areas involved in self-control
October 20, 2018 - KHN’s ‘What the Health?’ Republicans’ preexisting political problem
October 20, 2018 - Research provides a more complete picture of suffering caused by terrorist attacks
October 20, 2018 - Eradicating Helicobacter pylori infections may be a key treatment for Parkinson’s disease
October 20, 2018 - Breast Cancer as a Dynamic Disease
October 20, 2018 - University of Pittsburgh wins NSF grant for big data research to prevent complications from anesthesia
October 20, 2018 - Skin-to-skin contact may promote attachment between parents and preterm infants
October 20, 2018 - Recommendations Developed to Verify NGT Placement in Children
October 20, 2018 - Weight loss can be boosted fivefold thanks to novel mental imagery technique
October 20, 2018 - Children with autism are more likely to be overweight, obese
October 20, 2018 - Nurses making conscientious objections to ethically-relevant policies lack support
October 20, 2018 - Prion strain diversity may be greater than previously thought
October 20, 2018 - Antidepressant treatment may lead to improvements in sleep quality of patients with depression
October 20, 2018 - Study reports increased risk of death in children with inflammatory bowel disease
October 20, 2018 - Number of Autism Genes Now Tops 100
October 20, 2018 - Total diet replacement programmes are effective for treating obesity
October 20, 2018 - CLARIOstar used for fluorescence measurements on CSIRO’s purpose-built research vessel
October 20, 2018 - People with more copies of AMY1 gene digest starchy carbohydrates faster
October 20, 2018 - Case Comprehensive Cancer Center wins NIH grant to study health disparities
October 20, 2018 - Newly discovered compound shows potential for treating Parkinson’s disease
October 20, 2018 - High rate of non-adherence to hormonal therapy found among premenopausal early breast cancer patients
October 20, 2018 - Immunotherapy medicine found to be effective in treating uveitis
October 20, 2018 - The Pistoia Alliance Calls for Greater Collaboration to Realise Benefits of Innovation and Announces Winners of the 2018 President’s Startup Challenge
October 20, 2018 - Female internists consistently earn less than men
October 20, 2018 - Stanford team looks at dangers of teens’ vaping habits
October 20, 2018 - New approach to understanding cancers will accelerate development of better treatments
October 20, 2018 - LJI and UC San Diego awarded $ 4.5 million as part of NCI’s Cancer Moonshot initiative
October 20, 2018 - School-based HPV vaccination did not increase risky sexual behaviors among adolescent girls
October 20, 2018 - Eye discovery to pave way for more successful corneal transplants
October 20, 2018 - New analysis examines the importance of location in the opioid crisis
October 20, 2018 - Green filters increase reading speed for children with dyslexia
October 19, 2018 - Bariatric Sx Cuts Macrovascular Complications in Obesity, T2DM
October 19, 2018 - Better assessments for early age-related macular degeneration
October 19, 2018 - Visible and valued: Stanford Medicine’s first-ever LGBTQ+ Forum | News Center
October 19, 2018 - Understanding of metal-free enzymes used by bacteria could lead to new effective antibiotics
October 19, 2018 - Beckman Coulter Life Sciences announces new research-focused website
October 19, 2018 - Study finds link between refined soluble fibers, gut microbiota and liver cancer
October 19, 2018 - Social media reduces risk of depression among seniors with pain
October 19, 2018 - Newly developed synthetic DNA molecule may one day be used as ‘vaccine’ for prostate cancer
October 19, 2018 - Preoperative weight loss may not provide health benefits after surgery
October 19, 2018 - U.S. Birth Rates Continue to Drop as Age of New Moms Rises
October 19, 2018 - New technology can keep an eye on babies’ movements in the womb
October 19, 2018 - Juul e-cigarettes pose addiction risk for young users | News Center
October 19, 2018 - Gene sequencing reveals crucial molecular aspects of Trypanosoma brucei
October 19, 2018 - New DNA vaccine strategy protects mice against lethal challenge by multiple H3N2 viruses
October 19, 2018 - Study shows close link between cytokine interleukin-1ß and obesity-promoted colon cancer
October 19, 2018 - Muscle mass plays a critical role in health, shows research
October 19, 2018 - Study finds undiagnosed prediabetes in many infertile men
October 19, 2018 - The Current issue of “The view from here” is concerned with Nanotherapeutic strategies
October 19, 2018 - Delay in replacing the Pap smear with HPV screening is costing lives
October 19, 2018 - Physicians battle pediatric diseases of ear, nose, throat in Zimbabwe | News Center
October 19, 2018 - Researchers investigate why some cancers affect only young women
October 19, 2018 - Drugmakers funnel millions to lawmakers; a few dozen get $100,000-plus
October 19, 2018 - Unselfish people tend to have more children and receive higher salaries
October 19, 2018 - New findings reveal potential cellular players in tumor microenvironment
October 19, 2018 - Study reveals impact of Juul use on teenagers and young adults
October 19, 2018 - Green leafy vegetables could help reduce macular degeneration risk
October 19, 2018 - Some countries take more time for reimbursement decisions on new cancer drugs
October 19, 2018 - Human brain cell transplant offers insights into neurological conditions
October 19, 2018 - Parental education associated with increased family health care spending
October 19, 2018 - New statistical method estimates long- and short-term risk of recurrence of breast cancer in US women
October 19, 2018 - Father’s exposure to nicotine may cause cognitive deficits in descendants
October 19, 2018 - Could we prevent Alzheimer’s disease by treating herpes?
October 19, 2018 - Nurse-led care can be more successful in managing gout
October 19, 2018 - Trump administration, pharma exchange verbal volleys on drug-price transparency
October 19, 2018 - Duke researchers find way to detect blood doping in athletes
October 19, 2018 - Many primary care doctors are still prescribing sedative drugs for older adults
FDA Approves Revcovi (elapegademase-lvlr) for Adenosine Deaminase Severe Combined Immune Deficiency (ADA-SCID)

FDA Approves Revcovi (elapegademase-lvlr) for Adenosine Deaminase Severe Combined Immune Deficiency (ADA-SCID)

image_pdfDownload PDFimage_print

FDA Approves Revcovi (elapegademase-lvlr) for Adenosine Deaminase Severe Combined Immune Deficiency (ADA-SCID)

GAITHERSBURG, Md.–(BUSINESS WIRE) October 05, 2018 –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.1

“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.

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.

ADVERSE REACTIONS

The most commonly reported adverse reactions were cough and vomiting.

In addition, the following post-marketing reports for the same class of enzyme replacement therapy used in the treatment of ADA-SCID, may also be seen with Revcovi treatment:

  • Hematologic events: hemolytic anemia, autoimmune hemolytic anemia, thrombocythemia, thrombocytopenia and autoimmune thrombocytopenia
  • Dermatological events: injection site erythema, urticaria
  • Lymphomas

IMPORTANT MONITORING INFORMATION

Treatment with Revcovi should be monitored by measuring trough plasma ADA activity and trough dAXP levels for maintenance of therapeutic targets. If a persistent decline in plasma ADA activity occurs, immune function and clinical status should be monitored closely, and precautions should be taken to minimize the risk of infection.

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.2,3 ADA-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.7

About 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.

1

Revcovi™ (elapegademase-lvlr) injection Prescribing Information. Leadiant Biosciences, Inc. 2017.

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.

Source: Leadiant Biosciences, Inc.

Posted: October 2018

Revcovi (elapegademase-lvlr) FDA Approval History

Tagged with:

About author

Related Articles