AVROBIO Receives Rare Pediatric Disease Designation from U.S. Food and Drug Administration (FDA) for First Gene Therapy in Development for Cystinosis
20 September 2022 - 9:00PM
Business Wire
Second AVROBIO gene therapy program that has
been granted rare pediatric disease designation by FDA
AVR-RD-04 has previously received orphan drug
designation from FDA and EMA
AVROBIO, Inc. (Nasdaq: AVRO), a leading clinical-stage gene
therapy company working to free people from a lifetime of genetic
disease, today announced that the U.S. Food and Drug Administration
(FDA) has granted rare pediatric disease designation to AVR-RD-04,
an investigational gene therapy for the treatment of cystinosis, a
life-threatening disease that causes progressive multi-organ
damage, including early, acute kidney disease progressing to
end-stage kidney disease.
FDA’s Rare Pediatric Disease Designation and Voucher Program is
intended to facilitate the development of new drugs and biologics
for the prevention and treatment of rare pediatric diseases.
Companies that receive approval for a New Drug Application (NDA) or
Biologics License Application (BLA) for a rare pediatric disease
may be eligible to receive a voucher for a priority review of a
subsequent marketing application for a different product. The
priority review voucher may be used by the company or sold to a
third party.
AVR-RD-04 is designed to genetically modify patients’ own
hematopoietic stem cells (HSCs) to express the gene encoding
cystinosin, the protein that is critically deficient in people
living with cystinosis.
Preliminary data from the ongoing University of California San
Diego Phase 1/2 clinical trial suggest that this approach is well
tolerated, with no adverse events (AEs) related to the drug product
reported to date. All AEs reported were related to myeloablative
conditioning, stem cell mobilization, underlying disease or
pre-existing conditions. The majority of AEs were mild or moderate
and resolved without clinical sequelae. Clinical data to date
indicate this investigational approach provides benefits in
multiple tissues evaluated, including the eyes, skin,
gastrointestinal mucosa and the neurocognitive system. The
collaborator-sponsored Phase 1/2 clinical trial is funded in part
by grants to University of California San Diego from the California
Institute for Regenerative Medicine (CIRM), Cystinosis Research
Foundation (CRF) and National Institutes of Health (NIH).
About Cystinosis Cystinosis is a rare, progressive
disease that impacts approximately 1,600 patients in the U.S.,
Europe and Japan and is marked by the accumulation of cystine in
cellular organelles known as lysosomes. Untreated cystinosis is
fatal at an early age. The current SOC for cystinosis, a treatment
regimen that can require dozens of pills per day, does not prevent
overall disease progression and carries side effects, such as
breath and body odor and gastrointestinal symptoms, which can
impede compliance. More than 90% of treated cystinosis patients
require a kidney transplant in the second or third decade of
life.
About AVROBIO Our vision is to bring personalized gene
therapy to the world. We target the root cause of genetic disease
by introducing a functional copy of the affected gene into
patients’ own hematopoietic stem cells (HSCs), with the goal to
durably express the therapeutic protein throughout the body,
including the central nervous system. Our first-in-class pipeline
includes clinical programs for cystinosis and Gaucher disease type
1, as well as preclinical programs for Gaucher disease type 3,
Hunter syndrome and Pompe disease. Our proprietary plato® gene
therapy platform is designed to be scaled to support late-stage
clinical development and commercialization globally. We are
headquartered in Cambridge, Mass. For additional information, visit
avrobio.com, and follow us on Twitter and LinkedIn.
Forward-Looking Statements This press release contains
forward-looking statements, including statements made pursuant to
the safe harbor provisions of the Private Securities Litigation
Reform Act of 1995. These statements may be identified by words and
phrases such as “aims,” “anticipates,” “believes,” “could,”
“designed to,” “estimates,” “expects,” “forecasts,” “goal,”
“intends,” “may,” “plans,” “possible,” “potential,” “seeks,”
“will,” and variations of these words and phrases or similar
expressions that are intended to identify forward-looking
statements. These forward-looking statements include, without
limitation, statements regarding our business strategy for and the
potential therapeutic benefits of our preclinical and clinical
product candidates, including AVR-RD-04 for the treatment of
cystinosis, the potential benefits and incentives provided by FDA’s
rare pediatric disease designation for AVR-RD-04, the design,
commencement, enrollment and timing of planned clinical trials,
preclinical or clinical trial results, product approvals and
regulatory pathways, our plans and expectations with respect to
interactions with regulatory agencies, anticipated benefits of our
gene therapy platform including potential impact on our
commercialization activities, timing and likelihood of success, the
expected benefits and results of our implementation of the plato
platform in our clinical trials and gene therapy programs, and the
expected safety profile of our preclinical and investigational gene
therapies. Any such statements in this press release that are not
statements of historical fact may be deemed to be forward-looking
statements. Results in preclinical or early-stage clinical trials
may not be indicative of results from later stage or larger scale
clinical trials and do not ensure regulatory approval. You should
not place undue reliance on these statements, or the scientific
data presented.
Any forward-looking statements in this press release are based
on AVROBIO’s current expectations, estimates and projections about
our industry as well as management’s current beliefs and
expectations of future events only as of today and are subject to a
number of risks and uncertainties that could cause actual results
to differ materially and adversely from those set forth in or
implied by such forward-looking statements. These risks and
uncertainties include, but are not limited to, the risk that any
one or more of AVROBIO’s product candidates will not be
successfully developed or commercialized, the risk of cessation or
delay of any ongoing or planned clinical trials of AVROBIO or our
collaborators, the risk that AVROBIO may not successfully recruit
or enroll a sufficient number of patients for our clinical trials,
the risk that AVROBIO may not realize the intended benefits of our
gene therapy platform, including the features of our plato®
platform, the risk that our product candidates or procedures in
connection with the administration thereof will not have the safety
or efficacy profile that we anticipate, the risk that prior
results, such as signals of safety, activity or durability of
effect, including beneficial effects seen in multiple organs and
tissues, observed from preclinical or clinical trials, will not be
replicated or will not continue in ongoing or future studies or
trials involving AVROBIO’s product candidates, the risk that we
will be unable to obtain and maintain regulatory approval for our
product candidates, the risk that the size and growth potential of
the market for our product candidates will not materialize as
expected, risks associated with our dependence on third-party
suppliers and manufacturers, risks regarding the accuracy of our
estimates of expenses and future revenue, risks relating to our
capital requirements and needs for additional financing, risks
relating to clinical trial and business interruptions resulting
from the COVID-19 outbreak or similar public health crises,
including that such interruptions may materially delay our
enrollment and development timelines and/or increase our
development costs or that data collection efforts may be impaired
or otherwise impacted by such crises, and risks relating to our
ability to obtain and maintain intellectual property protection for
our product candidates. For a discussion of these and other risks
and uncertainties, and other important factors, any of which could
cause AVROBIO’s actual results to differ materially and adversely
from those contained in the forward-looking statements, see the
section entitled “Risk Factors” in AVROBIO’s most recent Annual or
Quarterly Report, as well as discussions of potential risks,
uncertainties and other important factors in AVROBIO’s subsequent
filings with the Securities and Exchange Commission. AVROBIO
explicitly disclaims any obligation to update any forward-looking
statements except to the extent required by law.
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version on businesswire.com: https://www.businesswire.com/news/home/20220920005367/en/
Investors: Christopher F. Brinzey ICR Westwicke
339-970-2843 chris.brinzey@westwicke.com
Media: Kit Rodophele Ten Bridge Communications
617-999-9620 krodophele@tenbridgecommunications.com
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