OPKO Health, Inc., a multinational biopharmaceutical and diagnostics company, announced the US Food and Drug Administration (FDA) has begun review of the Company’s New Drug Application (NDA) for calcifediol modified-release capsules, indicated for the prevention and treatment of secondary hyperparathyroidism in patients diagnosed with Stage 3 or 4 chronic kidney disease (CKD) with accompanying vitamin D deficiency.
“FDA acceptance of this NDA should be cheered by healthcare professionals who care for CKD patients,” said Dr. Kevin J. Martin, Director of the Division of Nephrology at Saint Louis University School of Medicine. “If approved, Rayaldee will provide a new therapeutic option for controlling elevated parathyroid hormone levels in this large and undertreated patient population.”
OPKO Health’s NDA application is backed by the findings of 3 randomized, double-blind, placebo-controlled clinical trials, and 1 open-label extension study which involved a target population across 105 sites in the country. These clinical studies successfully met all set primary efficacy and safety endpoints.
The proposed trade name for OPKO Health’s product candidate is Rayaldee. It is an orally stable vitamin D prohormone treatment that features a proprietary modified-release formulation that works to gradually restore blood levels of 25-hydroxyvitamin D (prohormone) to a target level of at least 30 ng/mL, without risk of upregulation of CYP24A1, a cytochrome P-450 enzyme known to inhibit the natural parathyroid hormone (PTH)-lowering effect. This therapeutic regulation helps prevent the upregulation of serum calcium and consequent vascular and renal calcification.
In a previous report on CKD, a team of researchers found that patients with chronic kidney disease (CKD) are more prone to develop gout later in life. Results revealed that there were 371 incident cases (231 men and 140 women) of gout over the follow-up of 140 421 person-years. The rates of gout incidence per 1,000 person-years for participants with and without CKD were of 6.82 and 2.43, respectively. Results from the multivariable Cox models showed that CKD was associated with gout, with a HR of 1.88 among men and 2.31 among women. Additional analyses using alternate definitions for CKD and cross-sectional study did not change the results.