Adverum Biotechnologies Granted Orphan Drug Designation by FDA for Gene Therapy Candidate in Preclinical Development for the Treatment of Blue Cone Monochromacy
05 January 2022 - 12:00AM
Adverum Biotechnologies, Inc. (Nasdaq: ADVM), a clinical-stage gene
therapy company targeting unmet medical needs in ocular and rare
diseases, announced the U.S. Food and Drug Administration (FDA) has
granted Orphan Drug Designation to ADVM-062 (AAV.7m8-L-opsin), a
novel gene therapy product candidate being developed as a potential
single intravitreal (IVT) administration for blue cone monochromacy
(BCM) by delivering a functional copy of the OPN1LW gene.
“We are excited to introduce ADVM-062, which builds on our
ophthalmology and gene therapy expertise to address the significant
unmet medical needs of patients living with blue cone monochromacy.
We are pleased the FDA has recognized the patient need by granting
Orphan Drug Designation and we look forward to advancing ADVM-062
towards the clinic and generating important data,” stated Laurent
Fischer, M.D., president and chief executive officer of Adverum
Biotechnologies.
BCM affects approximately 1 to 9 in 100,000 males, worldwide.
This X-linked recessive hereditary condition is caused by mutations
in either the L or the M opsin gene(s) and can manifest in loss of
visual acuity, photophobia, myopia and infantile nystagmus that can
persist into adulthood. Consequently, individuals with BCM have
visual impairments to important aspects of daily living such as
facial recognition, learning, reading, and daylight vision.
Currently, there is no cure for BCM.
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 or disorders that affect fewer than 200,000 people in the
United States. 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-062
should the company receive FDA approval for the treatment
of BCM for this product candidate.
Anticipated Milestones for ADVM-062
- Plan to present preclinical data supporting proof of concept of
ADVM-062 in 1H2022.
- Plan to submit an Investigational New Drug (IND) application by
year-end 2022.
About ADVM-062 Gene Therapy
ADVM-062 is a gene therapy candidate specifically designed to
deliver a functional copy of the OPN1LW gene to the foveal cones of
patients suffering from BCM via a single IVT injection. ADVM-062
utilizes Adverum’s propriety vector capsid,
AAV.7m8.
About Adverum Biotechnologies
Adverum Biotechnologies (Nasdaq: ADVM) is a clinical-stage gene
therapy company targeting unmet medical needs in serious ocular and
rare diseases. Adverum’s lead gene therapy clinical candidate,
ADVM-022, is being evaluated as a one-time, intravitreal injection
for the treatment of patients with neovascular or wet age-related
macular degeneration (wet AMD). For more information, please visit
www.adverum.com.
Forward-looking Statements
Statements contained in this press release regarding 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
the statements under the caption “Anticipated Milestones for
ADVM-062.” Actual results could differ materially from those
anticipated in such forward-looking statements as a result of
various risks and uncertainties, including risks inherent to,
without limitation: Adverum’s novel technology, which makes it
difficult to predict the time and cost of product candidate
development and regulatory uncertainties; and other risks and
uncertainties facing Adverum described more fully in Adverum’s Form
10-Q filed with the SEC on November 4, 2021, under the
heading “Risk Factors.” 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.
Inquiries Anand ReddiVice President, Head of
Corporate Strategy and External Affairs & EngagementAdverum
Biotechnologies, Inc.T: 650-649-1358
Or
Laurence WattsGilmartin GroupT: 619-916-7620E:
laurence@gilmartinir.com
Source: Adverum Biotechnologies, Inc.
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