Organovo Receives Orphan Designation From U.S. FDA for 3D Bioprinted Therapeutic Liver Tissue Treatment of Alpha-1 Antitrypsi...
December 26 2017 - 8:05AM
Organovo Holdings, Inc. (NASDAQ:ONVO) (“Organovo”) today announced
that the U.S. Food and Drug Administration (“FDA”) granted orphan
drug designation for the Company’s treatment of alpha-1 antitrypsin
deficiency (“A1AT”) with its 3D bioprinted liver therapeutic
tissue.
“We are extremely pleased to receive orphan designation for our
NovoTissues® treatment of A1AT,” said Taylor J. Crouch, CEO,
Organovo. “The FDA’s rapid action recognizes the importance
of developing regenerative medicine therapeutic applications, and
mirrors our own urgency in addressing this devastating
disease. With tens of thousands of patients being treated for
inborn errors of metabolism (“IEMs”) in the U.S., and an annual
cost per patient that exceeds $250,000 for drug therapy
alone, these patient populations are in desperate need of new
treatment options.”
Crouch concluded, “This is a critical milestone that supports
our ongoing development of 3D bioprinted tissues for therapeutic
use. We remain on track for filing an Investigational New
Drug (“IND”) application with the FDA in calendar-year 2020, as we
continue to conduct safety and dosing investigations in small
animal disease models and move to defining and scoping IND enabling
studies.”
The FDA Orphan Drug designation program provides incentives to
sponsors that are developing therapies for rare diseases which
affect fewer than 200,000 people in the United
States. Organovo is now qualified to receive significant
benefits throughout its orphan drug development program including
more frequent FDA interactions, protocol assistance, and tax
credits for clinical research costs. The designation also
includes a waiver of certain fees and a seven-year term of market
exclusivity upon FDA approval of the orphan drug, and can provide
for a more streamlined and cost-effective path through to
commercialization.
About Organovo Holdings, Inc.
Organovo designs and creates functional, three-dimensional human
tissues for use in drug discovery, clinical development, and
therapeutic applications. The Company develops 3D human
tissue systems through internal research programs and in
collaboration with pharmaceutical, academic and other
partners. Organovo's 3D human tissues have the potential to
transform the drug discovery process, enabling treatments to be
developed more effectively and with greater relevance to
performance in human trials and commercialization. The
Company’s ExViveTM Human Liver and Kidney Tissues are used in
high-value drug profiling, including compound screening in disease
models, toxicology, target and marker discovery/validation, and
other drug testing. The Company is also advancing a
preclinical program to develop its NovoTissues® liver therapeutic
tissues for critical unmet medical needs, including certain
life-threatening pediatric diseases. In addition to numerous
scientific publications, the Company’s technology has been featured
in The Wall Street Journal, Time Magazine, The Economist, Forbes,
and numerous other media outlets. Organovo is changing the
shape of life science research and transforming medical care.
Learn more at www.organovo.com.
Forward-Looking Statements
Any statements contained in this press release that do not
describe historical facts constitute forward-looking statements as
that term is defined in the Private Securities Litigation Reform
Act of 1995. Any forward-looking statements contained herein
are based on current expectations, but are subject to a number of
risks and uncertainties. The factors that could cause the
Company's actual future results to differ materially from current
expectations include, but are not limited to, risks and
uncertainties relating to the Company's ability to develop, market
and sell products and services based on its technology; the
expected benefits and efficacy of the Company's products, services
and technology; the Company’s ability to successfully complete
studies and provide the technical information required to support
market acceptance of its products, services and technology, on a
timely basis or at all; the Company's business, research, product
development, regulatory approval, marketing and distribution plans
and strategies, including its use of third party distributors; the
Company's ability to secure additional contracted collaborative
relationships; the final results of the Company's preclinical
studies may be different from the Company's studies or interim
preclinical data results and may not support further clinical
development of its therapeutic tissues; the Company may not
successfully complete the required preclinical and clinical trials
required to obtain regulatory approval for its therapeutic tissues
on a timely basis or at all; the Company’s ability to control the
costs and to achieve the expected operational benefits and long-
term cost savings of its previously announced restructuring plan;
and the Company’s ability to meet its fiscal year 2018 outlook.
These and other factors are identified and described in more detail
in the Company's filings with the SEC, including its Annual Report
on Form 10-K filed with the SEC on June 7, 2017. You should not
place undue reliance on these forward-looking statements, which
speak only as of the date that they were made. These cautionary
statements should be considered with any written or oral
forward-looking statements that the Company may issue in the
future. Except as required by applicable law, including the
securities laws of the United States, the Company does not intend
to update any of the forward-looking statements to conform these
statements to reflect actual results, later events or circumstances
or to reflect the occurrence of unanticipated events.
Investor Contact:
Steve Kunszabo
Organovo Holdings, Inc.
+1 (858) 224-1092
skunszabo@organovo.com
Press Contact:
Jessica Yingling
Little Dog Communications
+1 (858) 344-8091
jessica@litldog.com
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