FDA Approves Rayaldee for Chronic Kidney Disease with Secondary Hyperparathyroidism

FDA Approves Rayaldee for Chronic Kidney Disease with Secondary Hyperparathyroidism

The U.S. Food and Drug Administration (FDA) has recently approved OPKO Health’s Rayaldee (calcifediol) capsules for the treatment of secondary hyperparathyroidism (SHPT) in adults with stage 3 or 4 chronic kidney disease (CKD) and vitamin D insufficiency.

Rayaldee capsules are a modified-release formulation of 25-hydroxyvitamin D3 (calcifediol) developed as a new monotherapy.

Kevin J. Martin, director of research in the Division of Nephrology at Saint Louis University School of Medicine, said Rayaldee is an important new option for treating these particular patients.

“The great majority of SHPT cases in this patient population are associated with vitamin D insufficiency, a problem that Rayaldee can correct,” he said.

The drug gradually restores serum total 25-hydroxyvitamin D concentrations to (or beyond) the established minimum satisfactory level of 30 ng/mL. It has a potential application as an adjunctive therapy to vitamin D hormone replacement therapy for SHPT in patients with CKD.

“Rayaldee fills a large void in the current treatment options for SHPT in pre-dialysis patients,” said 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, and requires timely and effective treatment, Bishop said.

“FDA’s approval of Rayaldee represents an important milestone for OPKO,” said OPKO CEO and Chairman Dr. Phillip Frost in a press release. “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.”

Two double-blind, randomized and dose-ranging placebo-controlled clinical trials are complete and have met all primary efficacy and safety endpoints. At 26 weeks, treatment with Rayaldee achieved at least a 30 percent reduction in plasma intact parathyroid hormone (iPTH) in patients with stage 3 or 4 CKD with SHPT and vitamin D insufficiency, compared to those treated with a placebo.

More than 80 percent of patients treated with Rayaldee saw their vitamin D insufficiency corrected compared with fewer than 7 percent of patients treated with the placebo.

During Rayaldee treatment there was an increase by 0.1 mg/dL in the mean serum calcium levels and in phosphorus levels compared to the placebo treatment. But these changes were considered clinically irrelevant. There were no differences between patients with stage 3 CKD or stage 4 CKD in Rayaldee’s efficacy or safety.



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