July 28, 2015 1:41pm EDT
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MIAMI--(BUSINESS WIRE)--
OPKO Health, Inc. (NYSE: OPK) today announced that the U.S. Food and
Drug Administration (FDA) has accepted for review OPKO’s New Drug
Application (NDA) for calcifediol modified-release capsules (proposed
trade name, Rayaldee™). In the NDA, OPKO seeks approval of 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.
“FDA acceptance of this NDA should be cheered by healthcare
professionals who care for CKD patients,” commented Dr. Kevin J. Martin,
Director of the Division of Nephrology at Saint Louis University School
of Medicine. “If approved, Rayaldee will provide a new therapeutic
option for controlling elevated parathyroid hormone levels in this large
and undertreated patient population.”
OPKO expects written notification of NDA acceptance along with the
Prescription Drug User Fee Act (PDUFA) date in the FDA’s 74-Day letter,
which the Company expects to receive by mid-August 2015.
The NDA is supported by 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
Rayaldee (calcifediol modified-release capsules) is an 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 interferes with the desired parathyroid
hormone (PTH)-lowering effect. Gradual elevation of serum total
25-hydroxyvitamin D is intended to prevent excessive elevation of serum
calcium and related vascular and renal calcification.
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 .
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 the expected
receipt of the 74-Day letter from the FDA, the contents of the
74-Day Letter and our ability to address the same, the expected
PDUFA date and whether Rayaldee will be approved by the FDA, our ability
to successfully launch and commercialize Rayaldee, 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, market potential for Rayaldee, 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/20150728006530/en/
OPKO Health, Inc.
Charles W. Bishop, PhD,
CEO, Renal Division
or
Media
Rooney & Associates
Terry
Rooney, 212-223-0689
or
Rooney
& Associates
Marion Janic, 212-223-4017
Source: OPKO Health, Inc.
Released July 28, 2015
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