- Approval Based Upon Two Randomized,
Double Blind, Placebo Controlled Trials and an Open Label Extension
Study
- There are Approximately 9 Million
Patients in the U.S. with SHPT, Stage 3 or 4 CKD and Vitamin D
Insufficiency; Large Potential U.S. Market
- U.S. Launch Planned for 2H 2016
OPKO Health, Inc. (NYSE:OPK) announced that the U.S. Food
and Drug Administration (FDA) has approved RAYALDEE® (calcifediol)
extended release capsules for the treatment of secondary
hyperparathyroidism (SHPT) in adults with stage 3 or 4 chronic
kidney disease (CKD) and serum total 25-hydroxyvitamin D levels
less than 30 ng/mL. RAYALDEE is a patented extended release product
containing 30 mcg of a prohormone called calcifediol
(25-hydroxyvitamin D3).
“FDA’s approval of RAYALDEE represents an important milestone
for OPKO,” noted Dr. Phillip Frost, CEO and Chairman of OPKO.
"RAYALDEE is the first product to receive FDA approval for this
important indication and is one of OPKO’s many pharmaceutical
products being developed for significant medical problems which
will benefit from new treatment options.”
Results from two 26 week placebo controlled, double blind phase
3 trials demonstrated that a larger proportion of stage 3 or 4 CKD
patients with SHPT and vitamin D insufficiency achieved ≥30%
reductions in plasma intact parathyroid hormone (iPTH) when treated
with RAYALDEE than with placebo. Vitamin D insufficiency was
corrected in more than 80% of the patients receiving RAYALDEE
compared with less than 7% of subjects receiving placebo. Mean
serum calcium and phosphorus levels increased by 0.1 mg/dL during
RAYALDEE treatment compared to placebo treatment, but these changes
were deemed clinically irrelevant. No differences in RAYALDEE’s
efficacy or safety were observed between patients with stage 3 CKD
or stage 4 CKD.
“RAYALDEE fills a large void in the current treatment options
for SHPT in predialysis patients,” commented Dr. Charles W. Bishop,
CEO of OPKO’s Renal Division. “The current standard of care is high
dose vitamin D supplementation, an approach for treating SHPT that
is neither FDA approved nor demonstrated to be safe and effective
in this population. SHPT is a progressive disease that becomes
increasingly debilitating and difficult to treat, necessitating
timely and effective treatment.”
“RAYALDEE is an important new option for treating SHPT in
patients with stage 3 or 4 CKD and vitamin D insufficiency,” stated
Kevin J. Martin, Director of Research, Division of Nephrology at
Saint Louis University School of Medicine. “The great majority of
SHPT cases in this patient population are associated with vitamin D
insufficiency, a problem that RAYALDEE can correct.”
About RAYALDEE
RAYALDEE (calcifediol) extended release capsules are approved by
the U.S. Food and Drug Administration (FDA) for the treatment of
SHPT in adult patients with stage 3 or 4 CKD and serum total
25-hydroxyvitamin D levels less than 30 ng/mL. RAYALDEE has a
patented formulation designed to raise serum total
25-hydroxyvitamin D (prohormone) concentrations to targeted levels
(at least 30 ng/mL) and to reduce elevated iPTH. OPKO expects to
launch RAYALDEE in the U.S. through its dedicated renal sales force
in the second half of 2016. RAYALDEE is not indicated in patients
with stage 5 chronic kidney disease or end-stage renal disease on
dialysis. The full prescribing information for RAYALDEE will be
available at www.opkorenal.com.
Potential side effects of RAYALDEE include hypercalcemia
(elevated serum calcium), which can also lead to digitalis
toxicity, and adynamic bone disease with subsequent increased risk
of fractures if intact PTH levels are suppressed by RAYALDEE to
abnormally low levels. Severe hypercalcemia may require emergency
attention; symptoms of hypercalcemia may include feeling tired,
difficulty thinking clearly, loss of appetite, nausea, vomiting,
constipation, increased thirst, increased urination, and weight
loss. Digitalis toxicity can be potentiated by hypercalcemia of any
cause. Excessive administration of RAYALDEE can cause
hypercalciuria, hypercalcemia, hyperphosphatemia, or
oversuppression of intact PTH. Common symptoms of vitamin D
overdosage may include constipation, decreased appetite,
dehydration, fatigue, irritability, muscle weakness, or vomiting.
Patients concomitantly taking cytochrome P450 inhibitors,
thiazides, cholestyramine, phenobarbital or other anticonvulsants
may require dose adjustments and more frequent monitoring.
The most common adverse reactions in clinical trials (≥3% and
more frequent than placebo) were anemia, nasopharyngitis, increased
blood creatinine, dyspnea, cough, congestive heart failure and
constipation.
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. Rayaldee is only indicated for treating SHPT in patients
with stage 3 or stage 4 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 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 25D. 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 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, an FDA approved
treatment for SHPT in stage 3-4 CKD patients with vitamin D
insufficiency, and VARUBI™ for chemotherapy induced nausea and
vomiting (oral formulation launched by partner Tesaro and IV
formulation PDUFA date: January 2017). 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 (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 RAYALDEE, our ability to successfully launch and
commercialize RAYALDEE and our other products under development,
expectations about RAYALDEE and its market size and potential, that
RAYALDEE will effectively control SHPT in patients with stage 3 or
4 CKD by correcting vitamin D insufficiency, that RAYALDEE fills a
large void in the current treatment options for SHPT in predialysis
patients, whether RAYALDEE will be highly effective in correcting
vitamin D insufficiency, and that we will be able to successfully
launch sales of RAYALDEE in the second half of 2016. 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 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.
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version on businesswire.com: http://www.businesswire.com/news/home/20160620006515/en/
OPKO Health, Inc.:Charles W. Bishop, 305-575-4100PhD,
CEO, Renal DivisionorMedia:Rooney & AssociatesTerry
Rooney, 212-223-0689trooney@rooneyco.comorMarion Janic,
212-223-4017mjanic@rooneyco.comorInvestors:LHAAnne Marie
Fields, 212-838-3777afields@lhai.comorBruce Voss,
310-691-7100bvoss@lhai.com
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