Adverum Biotechnologies Receives Orphan Drug Designation for ADVM-053, a Novel Gene Therapy Candidate for the Treatment of He...
August 24 2018 - 7:30AM
- ADVM-053 is a novel gene therapy candidate
designed as a potential single‑administration treatment for
hereditary angioedema (“HAE”)- Plans to submit an Investigational
New Drug (IND) application to the FDA for ADVM-053 in 4Q18
Adverum Biotechnologies, Inc. (Nasdaq: ADVM), a clinical-stage gene
therapy company targeting unmet medical needs in serious rare and
ocular diseases, today announced that the United States (U.S.) Food
and Drug Administration (FDA) has granted Orphan Drug Designation
(ODD) to ADVM-053, a preclinical gene therapy candidate being
investigated as a potential single‑administration treatment which
has the potential to provide sustained levels of the C1 esterase
inhibitor (“C1EI”) protein.
HAE affects approximately 8,000 individuals in the U.S. This
disease is caused by a genetic mutation that results in low levels
of C1 esterase inhibitor which can be associated with sudden
swelling and edema of respiratory airways, gastrointestinal tract,
and extremities.
“We are pleased to receive the Orphan Drug Designation for
ADVM-053 from the FDA," said Leone Patterson, interim president and
chief executive officer of Adverum Biotechnologies. “We are
committed to developing effective treatments for patients living
with HAE and the support from the FDA will be invaluable towards
this goal. We look forward to submitting our IND application in the
fourth quarter.”
Orphan drug designation is granted by the FDA to novel drugs and
biologics, which are defined as those intended for the safe and
effective treatment, diagnosis or prevention of rare
diseases/disorders that affect fewer than 200,000 people in the
U.S. The designation provides incentives for sponsors to develop
products for rare diseases, which may include tax credits towards
the cost of clinical trials and prescription drug user fee waivers.
The orphan drug designation also could entitle Adverum
Biotechnologies to a seven-year period of marketing exclusivity in
the United States for ADVM-053 should the company receive FDA
approval for the treatment of HAE for this product candidate.
About ADVM-053ADVM-053 (AAVrh.10-C1EI) is
designed as a single‑administration treatment with the potential to
provide sustained expression of the C1 esterase inhibitor protein
to eliminate protein level variability and to prevent breakthrough
angioedema attacks. In preclinical studies, a single intravenous
administration of ADVM-053 increased C1EI protein expression above
therapeutic levels and decreased vascular permeability in a mouse
model of HAE.
About Adverum Biotechnologies,
Inc.Adverum is a clinical-stage gene therapy company
targeting unmet medical needs in serious rare and ocular diseases.
Adverum has a robust pipeline that includes product candidates
designed to treat rare diseases alpha-1 antitrypsin (A1AT)
deficiency and hereditary angioedema (HAE) as well as wet
age-related macular degeneration (wAMD). Leveraging a
next-generation adeno-associated virus (AAV)-based directed
evolution platform, Adverum generates product candidates designed
to provide durable efficacy by inducing sustained expression of a
therapeutic protein. Adverum has collaboration agreements with
Regeneron Pharmaceuticals to research, develop, and commercialize
gene therapy products for ophthalmic diseases and Editas Medicine
to explore the delivery of genome editing medicines for the
treatment of inherited retinal diseases. Adverum’s core
capabilities include clinical development and in-house
manufacturing expertise, specifically in process development and
assay development. For more information please visit
www.adverum.com.
Statements contained in this press release regarding matters
events or results that may occur in the future are “forward-looking
statements” within the meaning of the Private Securities Litigation
Reform Act of 1995. Such statements include, but are not limited
to, statements regarding Adverum’s plans to submit an IND
Application for ADVM-053 for HAE in the fourth quarter of 2018, all
of which are based on certain assumptions made by Adverum on
current conditions, expected future developments and other factors
Adverum believes are appropriate in the circumstances. Adverum may
not consummate any of these plans or these product, clinical
development or regulatory goals in a timely manner, or at all, or
otherwise carry out the intentions or meet the expectations or
projections disclosed in its forward-looking statements, and you
should not place undue reliance on these forward-looking
statements. Actual results and the timing of events could differ
materially from those anticipated in such forward-looking
statements as a result of various risks and uncertainties, which
include, without limitation, the risk of a delay in the enrollment
of patients in Adverum’s clinical studies or in the manufacturing
of products to be used in such clinical studies, as well as the
risks and uncertainties facing Adverum described more fully in
Adverum’s periodic reports filed with the Securities and Exchange
Commission (SEC), especially under the caption “Risk Factors” in
its latest Quarterly Report on Form 10-Q filed with the SEC on
August 8, 2018. All forward-looking statements contained in this
press release speak only as of the date on which they were made.
Adverum undertakes no obligation to update such statements to
reflect events that occur or circumstances that exist after the
date on which they were made.
Contact for Adverum:
Katherine Bock
Vice President Investor Relations
& Corporate Communications
650-656-9347
kbock@adverum.com
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