June 21, 2016 2:00am EDT
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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
MIAMI--(BUSINESS WIRE)--
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 D 3 ).
“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 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/20160620006515/en/
Charles W. Bishop, 305-575-4100
PhD,
CEO, Renal Division
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 June 21, 2016
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