May 29, 2015 2:42pm EDT
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MIAMI--(BUSINESS WIRE)--
OPKO Health, Inc. (NYSE: OPK), today announced that it has submitted a
New Drug Application (NDA) for oral Rayaldee to the U.S. Food and Drug
Administration (FDA). The NDA requests marketing approval for Rayaldee
for the prevention and treatment of secondary hyperparathyroidism (SHPT)
in patients with stage 3 or 4 chronic kidney disease (CKD) and vitamin D
insufficiency.
“The submission of this NDA represents a significant milestone in the
development of Rayaldee, and it underscores our strong commitment to
making improved therapies available to patients who suffer from chronic
kidney disease,” commented Dr. Phillip Frost, CEO and Chairman of OPKO.
“We believe Rayaldee will be the first product to receive FDA approval
for this important indication.”
The Rayaldee application is supported by positive data from three
randomized, double-blind, placebo-controlled studies and one open-label
extension study conducted in the targeted patient population at a total
of 105 U.S. sites. These studies met all primary efficacy and safety
endpoints, as previously announced.
About Rayaldee TM
Rayaldee is a first-in-class oral vitamin D prohormone treatment being
developed for SHPT in patients with stage 3 or 4 CKD and vitamin D
insufficiency. It has a proprietary modified-release formulation
designed to gradually and reliably raise serum total 25-hydroxyvitamin D
(prohormone) concentrations to targeted levels (at least 30 ng/mL) while
avoiding upregulation of CYP24A1, a cytochrome P-450 enzyme which
reduces the desired parathyroid hormone (PTH)-lowering efficacy. Gradual
elevation of serum total 25-hydroxyvitamin D prevents excessive
elevation of serum calcium and related vascular and renal calcification.
Such side effects limit the value of current vitamin D hormone
therapies. Rayaldee is expected to address the approximately 20 million
patients in the U.S., and many more elsewhere, with stage 3 or 4 CKD,
SHPT and vitamin D insufficiency.
About Chronic Kidney Disease
CKD is a condition characterized by a progressive decline in kidney
function. The kidney is normally responsible for excreting waste and
excess water from the body, and for regulating various hormones. CKD is
classified in five stages — mild (stage 1) to severe (stage 5) disease —
as measured by the kidney's glomerular filtration rate. According to the
National Kidney Foundation, CKD afflicts over 26 million people in the
U.S., including more than 20 million patients with moderate (stages 3 or
4) and severe (stage 5) forms of CKD. In stage 5 CKD, kidney function is
minimal to absent and patients require regular dialysis or a kidney
transplant for survival.
About Vitamin D Insufficiency
Vitamin D insufficiency is a condition in which the body has low vitamin
D stores, characterized by inadequate blood levels of vitamin D
prohormone, known as 25-hydroxyvitamin D. An estimated 70-90% of CKD
patients have vitamin D insufficiency, which can lead to SHPT and
resultant debilitating bone diseases. Vitamin D insufficiency has been
associated with increased mortality in CKD.
About Secondary Hyperparathyroidism (SHPT)
SHPT is a condition commonly associated with CKD in which the
parathyroid glands secrete excessive amounts of PTH. SHPT arises as a
result of vitamin D insufficiency or impaired kidney function that
prevents sufficient production of vitamin D hormone to properly regulate
calcium and phosphorus metabolism, and PTH secretion. Prolonged
elevation of blood PTH causes excessive calcium and phosphorus to be
released from bone, leading to elevated serum calcium and phosphorus,
softening of the bones (osteomalacia) and calcification of vascular and
renal tissues. SHPT affects 40-60% of patients with moderate CKD and
approximately 90% of patients with severe CKD. Vitamin D therapy for
SHPT is associated with reduced mortality in CKD patients.
About OPKO
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 .
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 ability to
obtain approval for, successfully launch and commercialize proprietary
renal disease products, including Rayaldee, that the NDA will be
accepted or approved, expectations about Rayaldee, that Rayaldee will
effectively control secondary hyperparathyroidism in patients with stage
3 or 4 chronic kidney disease by correcting vitamin D insufficiency,
whether Rayaldee is appropriate for patients with minimal functioning
kidney mass and its efficacy during longer term administration, whether
Rayaldee will be highly effective in correcting vitamin D insufficiency,
allowing more reliable treatment of patients, whether it is the solution
to secondary hyperparathyroidism associated with vitamin D insufficiency
for the 20 million pre-dialysis CKD patients in the U.S. and elsewhere,
market potential for Rayaldee, that Rayaldee will treat vitamin D
insufficiency and gradually correct elevated PTH, without safety
concerns, and that we will be able to successfully develop, obtain
approval for and launch sales of Rayaldee. 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 the phase
3 clinical trials for Rayaldee 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
Rayaldee, and that Rayaldee may not have advantages or prove to be
superior over presently marketed products, including the currently used
high monthly doses of prescription vitamin D 2 ,
activated vitamin D hormone and over-the-counter vitamin D supplements .
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.
View source version on businesswire.com: http://www.businesswire.com/news/home/20150529006010/en/
OPKO Health, Inc.
Steven D. Rubin, 305-575-4100
or
Charles
Bishop 305-575-4105
Source: OPKO Health, Inc.
Released May 29, 2015
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