February 28, 2014 9:58am EST
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MIAMI--(BUSINESS WIRE)--
OPKO Health, Inc. (NYSE:OPK) today announced that the U.S. Food and Drug
Administration (FDA) has granted orphan drug designation to OPKO's
longer-acting version of clotting Factor VIIa (Factor VIIa-CTP) for the
treatment of bleeding episodes in patients with hemophilia A or B with
inhibitors to Factor VIII or Factor IX. These patients are currently
being treated by commercially-available Factor VIIa, with estimated 2013
worldwide sales of $1.7 billion.
Currently, Factor VIIa therapy is available only as an intravenous (IV)
formulation, which requires multiple injections to treat a bleeding
episode due to Factor VIIa’s short half-life. In addition, the
requirement for multiple weekly injections can be onerous for patients
interested in preventive prophylactic treatment of the disease,
especially children.
Preclinical data presented by OPKO at the 7th Annual Congress of the
European Association for Haemophilia and Allied Disorders (EAHAD) on
February 26-28, 2014 in Brussels show that OPKO’s long-acting Factor
VIIa-CTP demonstrated the potential for substantial improvement of the
quality of life of patients, via both IV and subcutaneous (SC)
administration. The combination of a long-acting Factor VIIa coupled
with SC administration using a simple injection could potentially change
the Factor VIIa market segment, allowing children and adults with
hemophilia to easily self-administer the drug at home on a prophylactic
basis.
Dr. Phillip Frost, CEO of OPKO, commented, “We are pleased with the new
orphan drug designation for Factor VIIa-CTP in the U.S., as it would
allow OPKO seven years of marketing exclusivity upon commercialization
of the drug. We have previously presented data in animal models of
hemophilia showing that Factor Vlla-CTP demonstrates superiority across
key efficacy and safety parameters compared to current Factor VIIa
therapy. We are enthused about the potential for SC administration due
to its improved bioavailability compared to presently available Factor
VIIa IV therapy. Expecting to initiate a Phase IIa trial with our IV
formulation in hemophilia patients in late 2014, we believe that we are
in a unique position to be first to market with a longer acting Factor
VIIa product.”
FDA's Orphan Drug Act of 1983 was designed to encourage the development
of products that demonstrate promise for the diagnosis, prevention
and/or treatment of life-threatening or very serious conditions that are
rare and affect 200,000 persons or less in the U.S. The orphan drug
designation provides for seven years of market exclusivity, reduction in
regulatory fees, certain tax credits and additional regulatory support
for R&D initiatives.
ABOUT OPKO HEALTH, INC.
OPKO is a multinational biopharmaceutical and diagnostics company that
seeks to establish industry-leading positions in large, rapidly growing
markets by leveraging its discovery, development and commercialization
expertise and novel and proprietary technologies. For more information,
visit http://www.opko.com .
SAFE HARBOR STATEMENT
This press release contains "forward-looking statements," as that term
is defined under the Private Securities Litigation Reform Act of 1995
(PSLRA), regarding product development efforts and other non-historical
facts about our expectations, beliefs or intentions regarding our
business, technologies and products, financial condition, strategies or
prospects, including statements regarding our expectations about our
inhibitors to Factor VIII or Factor IX, whether Factor VIIa-CTP has the
potential for substantial improvement of the quality of life of
patients, via both IV and subcutaneous (SC) administration, whether
Factor VIIa-CTP will be able to be administered subcutaneously using a
simple injection and allow children and adults with hemophilia to easily
self-administer the drug at home on a prophylactic basis, whether we
will be able to initiate a Phase IIa trial with our IV formulation in
hemophilia patients in late 2014, whether we will be in a position to be
first to market with a longer acting Factor VIIa product, expectations
about market potential for Factor VIIa-CTP, whether we will be able to
successfully develop, obtain approval for and launch sales of the Factor
VIIa-CTP, and the expected completion dates for our trials. Many factors
could cause our actual activities or results to differ materially from
the activities and results anticipated in forward-looking statements.
These factors include those described in our filings with the Securities
and Exchange Commission, as well as risks inherent in funding,
developing and obtaining regulatory approvals of new,
commercially-viable and competitive products and treatments, including
the risks that clinical trials for Factor VIIa-CTP may not be successful
or achieve the expected results or effectiveness, and may not generate
data that would support the approval or marketing of this product for
the indications being studied, that others may develop products which
are superior to Factor VIIa-CTP, and that Factor VIIa-CTP may not have
advantages or prove to be superior over presently marketed products. In
addition, forward-looking statements may also be adversely affected by
general market factors, competitive product development, product
availability, federal and state regulations and legislation, the
regulatory process for new products and indications, manufacturing
issues that may arise, patent positions and litigation, among other
factors. The forward-looking statements contained in this press release
speak only as of the date the statements were made and we do not
undertake any obligation to update forward-looking statements. We intend
that all forward-looking statements be subject to the safe-harbor
provisions of the PSLRA.
OPKO Health, Inc .
Steven D. Rubin or Juan F. Rodriguez,
Source: OPKO Health, Inc.
Released February 28, 2014
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