FDA Approves New Drug Application for RAYALDEE® to Treat Secondary Hyperparathyroidism Associated with Vitamin D Insufficiency in Stage 3-4 Chronic Kidney Disease
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.
5 hours ago
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MIAMI--(BUSINESS WIRE)--
OPKO Health, Inc. (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.”Zac
