April 27, 2016 12:43pm EDT
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MIAMI--(BUSINESS WIRE)--
OPKO Health, Inc. (NYSE: OPK )
today announced the U.S. Food and Drug Administration (FDA) has accepted
OPKO’s resubmission on April 22, 2016 of the New Drug Application (NDA)
for RAYALDEE ® (calcifediol) for the treatment of secondary
hyperparathyroidism (SHPT) in patients with stage 3 or 4 chronic kidney
disease (CKD) and vitamin D insufficiency.
OPKO resubmitted the NDA following receipt of a complete response letter
(CRL) from the FDA on March 29, 2016, in which the FDA indicated the NDA
could not be approved due to deficiencies observed during a facility
inspection of OPKO’s third party manufacturer. The observations were not
specific to RAYALDEE manufacturing, and the CRL did not cite any safety,
efficacy or labeling issues with regard to RAYALDEE, nor did it request
any additional studies to be conducted prior to FDA approval.
A six month review period has been assigned for the resubmitted NDA, and
the new Prescription Drug User Fee Act (PDUFA) date will be October 22,
2016.
"We have worked closely with our third party manufacturer to ensure the
FDA’s inspection observations are promptly and fully addressed, and we
believe that our resubmission reflects OPKO’s strong commitment to
providing a new treatment for SHPT in CKD patients as soon as possible,”
stated Phillip Frost, M.D., Chairman and CEO of OPKO. “If approved,
RAYALDEE will be the first drug approved for this important indication.”
About RAYALDEE
RAYALDEE (calcifediol) extended-release capsules are pending approval by
the U.S. Food and Drug Administration (FDA) for the treatment of SHPT in
adult patients with stage 3 or 4 CKD and vitamin D insufficiency.
RAYALDEE has a proprietary formulation designed to raise serum total
25-hydroxyvitamin D (prohormone) concentrations to targeted levels (at
least 30 ng/mL) and to reduce elevated intact parathyroid hormone
(iPTH). RAYALDEE is presently expected to be launched in the U.S. by
OPKO's dedicated sales force in 4Q 2016.
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 parathyroid hormone
(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 Health, Inc.
OPKO Health, Inc. is a diversified healthcare company that seeks to
establish industry-leading positions in large, rapidly growing markets.
Our diagnostics business includes Bio-Reference Laboratories, the
nation's third-largest clinical laboratory with a core genetic testing
business and a 420-person sales force to drive growth and leverage new
products, including the 4Kscore® prostate cancer test and the Claros®1
in-office immunoassay platform. Our pharmaceutical business features
RAYALDEE, a treatment pending FDA approval for SHPT in stage 3-4 CKD
patients with vitamin D insufficiency, and VARUBI™ for
chemotherapy-induced nausea and vomiting (oral formulation approved by
FDA and launched by partner Tesaro, IV formulation in Phase 3). Our
biologics business includes hGH-CTP, a once-weekly human growth hormone
injection (in Phase 3 and partnered with Pfizer), and a long-acting
Factor VIIa drug for hemophilia (entering Phase 2a). We also have
production and distribution assets worldwide, multiple strategic
investments and an active business development strategy. More
information is available at 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 whether
inspection observations at our third party manufacturer have been fully
addressed, whether RAYALDEE will be approved by the FDA and the timing
thereof, the expected PDUFA date and launch date for RAYALDEE, our
ability to successfully launch and commercialize RAYALDEE, expectations
about RAYALDEE, and that RAYALDEE will effectively control secondary
hyperparathyroidism in patients with stage 3 or 4 chronic kidney disease
by correcting vitamin D insufficiency. 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 have generated 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 D2, 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/20160427006350/en/
OPKO Health, Inc.
Tara Mackay, 305-575-4100
Investor
Relations
or
Rooney & Associates
Terry
Rooney, 212-223-0689
or
Marion
Janic, 212-223-4017
or
LHA
Anne
Marie Fields, 212-838-3777
or
Bruce
Voss, 310-691-7100
Source: OPKO Health, Inc.
Released April 27, 2016
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