"FDA's approval of RAYALDEE represents an important milestone for OPKO,"
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.
"RAYALDEE is an important new option for treating SHPT in patients with
stage 3 or 4 CKD and vitamin D insufficiency," stated
RAYALDEE (calcifediol) extended release capsules are approved by the
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
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.
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
PhD, CEO, Renal Division
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