FDA Approves Secondary Hyperparathyroidism Treatment

The FDA has approved OPKO Health Inc’s calcifediol (Rayaldee) extended-release tablets for the treatment of secondary hyperparathyroidism (SHPT) in adults with stage 3 or 4 chronic kidney disease who are vitamin D deficient.

The FDA based its approval on 2 randomized, double-blind, placebo-controlled phase 3 studies and an open-label extension study.

In the 2 phase 3 studies, patients with SHPT and vitamin D deficiencies achieved at least 30% reductions in plasma intact parathyroid hormone compared with placebo. Vitamin D deficiency was also corrected in more than 80% of the study participants who used Rayaldee.

“Rayaldee fills a large void in the current treatment options for SHPT in predialysis patients,” said Charles W. Bishop, CEO of OPKO’s Renal Division, in a press release. “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 not indicated for use in patients with stage 5 chronic kidney disease.

Some of the adverse effects of the treatment include hypercalcemia, which may result in digitalis toxicity or adynamic bone disease. This can lead to an increased risk of fractures. The most common adverse effects are anemia, nasopharyngitis, and increased blood creatinine.