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

GAITHERSBURG, Md.–(BUSINESS WIRE)–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.

Please refer to Revcovi’s 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.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.

Contacts

Leadiant Biosciences, Inc.
Nancy Parsons, 301-670-2481
Vice
President, Pharmaceutical Products
nancy.parsons@leadiant.com
or
Media:
SmithSolve
LLC on behalf of Leadiant Biosciences, Inc.
Alex Van
Rees, 973-442-1555 ext. 111
alex.vanrees@smithsolve.com

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