Cystaran Shortage Notification – A Message to the Community

— Registry Study Will Provide Supplemental Information on Revcovi in a Post-Marketing Commitment Study —

Gaithersburg, MD – February 11, 2020Leadiant Biosciences, Inc. announced it is conducting the Registry Study of Revcovi® (elapegademase-lvlr) injection 1.6 mg/ml Treatment in Patients with Adenosine Deaminase Severe Combined Immune Deficiency (ADA-SCID). The registry study will follow pediatric and adult ADA-SCID patients for two years or longer to collect supplemental information on those being treated with Revcovi. Revcovi, an enzyme replacement therapy (ERT), was approved by the U.S. Food and Drug Administration (FDA) in October 2018 for the treatment of ADA-SCID in pediatric and adult patients. The most commonly reported adverse reactions are cough and vomiting. Please see the Important Safety Information below and refer to the Revcovi Full Prescribing Information for more details.

“ADA-SCID is an ultra-rare, life threatening genetic disorder. Fortunately, therapies exist, including enzyme replacement therapy with Revcovi, that alter the natural history of the disorder and provide support for the immune system,” said Joseph M. Wiley, M.D., F.A.A.P., Vice President, Medical Affairs, Drug Safety and Pharmacovigilance, Leadiant Biosciences, Inc. “Revcovi was approved based on evidence for safety and efficacy in pediatric and adult patients with ADA-SCID. Leadiant Biosciences is proud to announce the introduction of a patient registry study that will provide supplemental information extended across a larger number of affected individuals. This registry is open to all patients in the U.S. with ADA-SCID who receive Revcovi, even if they plan to move on to other treatments. The registry is another demonstration of the commitment of Leadiant Biosciences to obtain relevant, real-world information regarding treatment of this disorder building on several decades of a legacy of support for ADA-SCID.”

The registry study is open to patients up to 65 years of age who have been diagnosed with ADA-SCID and receive Revcovi as ERT.

“The information obtained through this registry study will provide additional knowledge and tools to help patients with this devastating disease. Through this ongoing research, we hope to better understand Revcovi, with the goal of benefitting patients in the future,” commented Jay Lieberman, MD, immunologist with LeBonheur Children’s Hospital and The University of Tennessee Health Sciences Center in Memphis. “We look forward to seeing the results from the study and working with Leadiant for the benefit of the ADA-SCID community.”

For more information about the Registry Study of Revcovi Treatment in Patients with ADA-SCID, visit www.clinicaltrials.gov and enter the identifier number NCT03878069.

About Revcovi
Revcovi (elapegademase-lvlr) is a PEGylated recombinant adenosine deaminase (rADA) enzyme indicated for the treatment of adenosine deaminase severe combined immune deficiency (ADA-SCID) in pediatric and adult patients. Revcovi works by supplementing levels of an essential enzyme called adenosine deaminase (ADA). In two multicenter trials, Revcovi supplemented ADA levels, reduced concentrations of toxic metabolites that are the hallmark of ADA-SCID, and improved total lymphocyte counts.

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.

Please refer to the Revcovi Full Prescribing Information.

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. ADA-SCID is typically diagnosed based on clinical symptoms within the first few months of life. The expansion of newborn screening now identifies most SCID patients (including ADA-SCID) before symptoms occur. If 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.

ADA-SCID results from mutations in the ADA gene, which provides instructions for producing the ADA enzyme. When functioning properly, ADA breaks down 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. However, mutations in the ADA gene reduce or eliminate the activity of adenosine deaminase, resulting in the buildup of adenosine and deoxyadenosine to toxic levels. The accumulation of these purine nucleotides is especially toxic to specialized lymphocytes called T-cells, B-cells and Natural Killer (NK) cells. The buildup of these toxic products in excess of normal causes the T-cells, B-cells and NK cells to die, leaving affected individuals with no significant immune defense and increasing their risk of infection.

ADA-SCID is estimated to occur in approximately one in 200,000 to one in 1,000,000 newborns around the world. The disorder is responsible for approximately 15% of SCID cases.

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 U.S. (Leadiant Biosciences, Inc.) and Europe (Leadiant Biosciences, Ltd.) For additional information, please visit leadiantbiosciences.com.

Contact at Leadiant Biosciences, Inc.:
Medical Affairs
leadiant@tmacmail.com
866-634-2765

Media
Alex Van Rees
SmithSolve LLC on behalf of Leadiant Biosciences, Inc.
Phone: 973-442-1555 ext. 111
alex.vanrees@smithsolve.com