Prevail Therapeutics’ PR001 Receives Orphan Drug Designation and Rare Pediatric Disease Designation from FDA
February 12 2020 - 6:00AM
Prevail Therapeutics Inc. (Nasdaq: PRVL) (“Prevail” or the
“Company”), a biotechnology company developing potentially
disease-modifying AAV-based gene therapies for patients with
neurodegenerative diseases, announced today that the U.S. Food and
Drug Administration (FDA) has granted Orphan Drug Designation for
the Company’s investigational gene therapy, PR001, for the
treatment of patients with Gaucher disease. The Company also
announced that the FDA has granted Rare Pediatric Disease
Designation for PR001 for the treatment of neuronopathic Gaucher
disease (nGD), the most severe form of the condition.
“We are pleased to receive these important designations from the
FDA, which underscore the critical nature of our work,” said Asa
Abeliovich, M.D., Ph.D., Founder and Chief Executive Officer of
Prevail. “These designations support our conviction that new gene
therapies for Gaucher disease are urgently needed — especially for
the severe, neuronopathic form of the disease, for which there are
no FDA-approved therapies.”
PR001 is also being developed as a potentially
disease-modifying, single-dose gene therapy for Parkinson’s disease
with GBA1 mutation (PD-GBA).
Orphan Drug Designation is granted by the FDA to drugs or
biologics intended to treat a rare disease or condition, defined as
one that affects fewer than 200,000 people in the United States.
Programs with Orphan Drug status receive partial tax credit for
clinical trial expenditures, waived user fees and eligibility for
seven years of marketing exclusivity.
Rare Pediatric Disease Designation is granted by the FDA in the
case of serious or life-threatening diseases affecting fewer than
200,000 people in the United States, primarily those 18 years of
age and younger. The sponsor of a drug with Rare Pediatric Disease
Designation may, upon marketing approval, qualify for receipt of a
priority review voucher applicable to a subsequent marketing
application, which voucher is fully transferable.
Prevail announced in December 2019 that its Investigational New
Drug (IND) application for PR001 for the treatment of nGD is
active. Prevail is proceeding with its Phase 1/2 clinical trial for
Type 2 Gaucher disease patients and expects to initiate patient
dosing during the first half of 2020. The Company also plans to
initiate a Phase 1/2 clinical trial for Type 3 Gaucher disease
patients in the second half of 2020 under the same IND.
About Neuronopathic Gaucher Disease Gaucher
disease is a lysosomal storage disorder caused by mutations in the
glucocerebrosidase gene GBA1, leading to multi-organ
pathology. Patients with severe mutations in
the GBA1 gene can present with neuronopathic Gaucher
disease, also termed Type 2 or Type 3 Gaucher disease. Type 2
Gaucher disease presents in infancy and involves rapidly
progressive neurodegeneration leading to death in infancy or early
childhood. Type 3 Gaucher disease typically presents in childhood
and can involve neurological manifestations such as seizures and
gaze and motor abnormalities. There are no therapies approved by
the FDA for the treatment of neuronopathic Gaucher
disease.
About Prevail TherapeuticsPrevail is a gene
therapy company leveraging breakthroughs in human genetics with the
goal of developing and commercializing disease-modifying AAV-based
gene therapies for patients with neurodegenerative diseases. The
company is developing PR001 for patients with Parkinson’s disease
with a GBA1 mutation (PD-GBA) and neuronopathic Gaucher disease;
PR006 for patients with frontotemporal dementia with GRN mutation
(FTD-GRN); and PR004 for patients with certain
synucleinopathies.
Prevail was founded by Dr. Asa Abeliovich in 2017, through a
collaborative effort with The Silverstein Foundation for
Parkinson’s with GBA and OrbiMed, and is headquartered in New York,
NY.
Forward-Looking Statements Related to
PrevailStatements contained in this press release
regarding matters that are not historical facts are
“forward-looking statements” within the meaning of the Private
Securities Litigation Reform Act of 1995, as amended. Examples of
these forward-looking statements include statements concerning
Prevail’s ability to develop meaningful therapeutic advances for
patients with neurodegenerative diseases, the timing and progress
of clinical trials, and the potential advantages of Orphan Drug
Designation and Rare Pediatric Disease Designation by the FDA.
Because such statements are subject to risks and uncertainties,
actual results may differ materially from those expressed or
implied by such forward-looking statements. These risks and
uncertainties include, among others: Prevail’s novel approach to
gene therapy makes it difficult to predict the time, cost and
potential success of product candidate development or regulatory
approval; Prevail’s gene therapy programs may not meet safety and
efficacy levels needed to support ongoing clinical development or
regulatory approval; the regulatory landscape for gene therapy is
rigorous, complex, uncertain and subject to change; despite
receiving Rare Pediatric Disease designation, the Company may not
be successful in obtaining a priority review voucher; gene
therapies are novel, complex and difficult to manufacture. These
and other risks are described more fully in Prevail’s filings with
the Securities and Exchange Commission (SEC), including the “Risk
Factors” section of the Company’s Quarterly Report on Form 10-Q for
the period ended September 30, 2019, filed with the SEC on November
12, 2019, and its other documents subsequently filed with or
furnished to the SEC. All forward-looking statements contained in
this press release speak only as of the date on which they were
made. Except to the extent required by law, Prevail undertakes no
obligation to update such statements to reflect events that occur
or circumstances that exist after the date on which they were
made.
Media Contact:Mary Carmichael Ten Bridge
Communications617-413-3543mary@tenbridgecommunications.com
Investor
Contact:investors@prevailtherapeutics.com
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