July 09, 2014 1:23pm EDT
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Broad Designations for Treatment of Hemophilia A & B with Inhibitors As
Well As Congenital Factor VII Deficiency
10-Year Marketing Exclusivity In Europe Upon Product Launch
Adds To Previously Granted Orphan Drug Designation in The U.S.
MIAMI--(BUSINESS WIRE)--
OPKO Health, Inc. (NYSE:OPK) today announced that the European Committee
for Orphan Medicinal Products (COMP) gave a positive opinion
recommending the approval of orphan drug designation for OPKO's
long-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 and congenital Factor VII
deficiency. These patients are currently being treated by
commercially-available Factor VIIa, with estimated 2013 worldwide sales
of $1.7 billion. Factor VIIa-CTP already obtained orphan status in the
U.S. earlier this year.
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 preventative prophylactic treatment of the disease,
especially children.
Preclinical data previously presented by OPKO show that OPKO’s
long-acting Factor VIIa-CTP has 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
would potentially change the Factor VIIa market segment, allowing
children and adults with hemophilia to be easily self-administered at
home on a prophylactic basis.
Dr. Phillip Frost, CEO of OPKO, commented, “We are pleased that the COMP
will recommend orphan designation for Factor VIIa-CTP in the EU, as it
would allow OPKO 10 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 of key efficacy and safety parameters compared to current
Factor VIIa products. We are excited about the potential for SC
administration. This may represent another major competitive advantage
for our long-acting Factor Vlla-CTP. We expect to initiate a Phase IIa
clinical 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 long acting Factor VII-a therapy.”
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 long-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, 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.
FTI Consulting
Barbara
Ryan, 212-850-5679
Managing Director
or
Media
FTI Consulting
Kimberly Ha, 212-850-5612
Senior
Director
Source: OPKO Health, Inc.
Released July 9, 2014
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