AGTC Announces U.S. FDA Orphan Drug Designation for Gene Therapy to Treat X-Linked Retinitis Pigmentosa
August 03 2017 - 04:01PM
Applied Genetic Technologies Corporation (NASDAQ:AGTC), a
biotechnology company conducting human clinical trials of
adeno-associated virus (AAV)-based gene therapies for the treatment
of rare diseases, today announced that the U.S. Food and Drug
Administration (FDA) has granted an orphan drug designation for its
gene therapy product candidate for the treatment of X-linked
retinitis pigmentosa (XLRP) caused by mutations in the RPGR gene.
In June 2016 the company announced that the European Commission
(EC) granted orphan medicinal product designation for the same
indication.
XLRP is an inherited condition that causes
progressive vision loss, beginning with night blindness in young
boys followed by progressive constriction of the field of vision.
Affected men become legally blind at an average of about 45 years
of age. The most common form of XLRP is caused by mutations in the
RPGR gene. Preclinical data indicate that treatment with a gene
therapy product slowed the loss of visual function in a canine
model of XLRP due to mutations in the RPGR gene. XLRP and X-linked
retinoschisis (XLRS) are the two development programs within AGTC's
collaboration and license agreement with Biogen Inc. (NASDAQ:BIIB),
to develop gene-based therapies for multiple ophthalmic
diseases.
“Receiving U.S. orphan drug designation for our
fourth gene therapy treatment candidate demonstrates our ongoing
commitment to addressing the unmet needs of people with rare
inherited diseases,” said Sue Washer, President and CEO of AGTC.
“XLRP affects approximately 20,000 people in the US and Europe. We
are on track to file an Investigational New Drug application for
this program this year, and this orphan drug designation will help
to accelerate the regulatory review process as we work to bring a
new treatment option to XLRP patients.”
AGTC has also been granted orphan drug
designation from the FDA and EC for its gene therapy product
candidates for the treatment of X-linked retinoschisis and for the
treatment of achromatopsia caused by mutations in the CNGA3 and
CNGB3 genes.
Orphan drug designation, covered by the U.S.
Orphan Drug Act of 1983, is granted to drugs or biologics that
treat a rare disease or condition affecting fewer than 200,000
individuals. Products receiving orphan drug designation are
eligible to receive market exclusivity for a period of seven years,
an accelerated regulatory review process, an exemption from certain
taxes and user fees and additional clinical support from FDA.
About AGTC
AGTC is a clinical-stage biotechnology company
that uses its proprietary gene therapy platform to develop products
designed to transform the lives of patients with severe diseases,
with an initial focus in ophthalmology. AGTC's lead product
candidates are designed to treat inherited orphan diseases of the
eye, caused by mutations in single genes that significantly affect
visual function and currently lack effective medical
treatments.
AGTC's product pipeline includes ophthalmology
programs in X-linked retinoschisis (XLRS), X-linked retinitis
pigmentosa (XLRP), achromatopsia, wet age-related macular
degeneration, and an optogenetics program with Bionic Sight. AGTC's
non-ophthalmology programs include its adrenoleukodystrophy program
and its otology program, which is in pre-clinical development, and
the company expects to advance several otology product candidates
into clinical development in the next few years. Each of AGTC's
XLRS, XLRP and adrenoleukodystrophy programs are part of its
collaboration and license agreement with Biogen. AGTC employs a
highly-targeted approach to selecting and designing its product
candidates, choosing to develop therapies for indications having
high unmet medical need that it believes are clinically feasible
and present commercial opportunities. AGTC has a significant
intellectual property portfolio and extensive expertise in the
design of gene therapy products including capsids, promoters and
expression cassettes, as well as, expertise in the formulation,
manufacture and physical delivery of gene therapy products.
Forward Looking Statements
This release contains forward-looking statements
that reflect AGTC's plans, estimates, assumptions and beliefs.
Forward-looking statements include information concerning possible
or assumed future results of operations, business strategies and
operations, preclinical and clinical product development and
regulatory progress, potential growth opportunities, potential
market opportunities and the effects of competition.
Forward-looking statements include all statements that are not
historical facts and can be identified by terms such as
"anticipates," "believes," "could," "seeks," "estimates,"
"expects," "intends," "may," "plans," "potential," "predicts,"
"projects," "should," "will," "would" or similar expressions and
the negatives of those terms. Actual results could differ
materially from those discussed in the forward-looking statements,
due to a number of important factors. Risks and uncertainties that
may cause actual results to differ materially include, among
others: no gene therapy products have been approved in the United
States and only two such products have been approved in Europe;
AGTC cannot predict when or if it will obtain regulatory approval
to commercialize a product candidate; uncertainty inherent in the
regulatory review process; risks and uncertainties associated with
drug development and commercialization; factors that could cause
actual results to differ materially from those described in the
forward-looking statements are set forth under the heading "Risk
Factors" in the Company's Annual Report on Form 10-K for the fiscal
year ended June 30, 2016, as updated by the Company’s Quarterly
Report on Form 10-Q for the fiscal quarter ended March 31, 2017, as
filed with the SEC. Given these uncertainties, you should not
place undue reliance on these forward-looking statements. Also,
forward-looking statements represent management's plans, estimates,
assumptions and beliefs only as of the date of this release. Except
as required by law, we assume no obligation to update these
forward-looking statements publicly or to update the reasons actual
results could differ materially from those anticipated in these
forward-looking statements, even if new information becomes
available in the future.
IR/PR CONTACTS:
David Carey (IR)
Lazar Partners Ltd.
T: (212) 867-1768
dcarey@lazarpartners.com
Lazar Partners for AGTC (PR)
T: (212) 867-1762
AGTC_PR@lazarpartners.com
CORPORATE CONTACTS:
Stephen Potter
Chief Business Officer
Applied Genetic Technologies Corporation
T: (617) 413-2754
spotter@agtc.com
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