OPKO Health announced top-line results from the final Phase 3 trial of Rayaldee for the treatment of secondary hyperparathyroidism (SHPT) in patients with Stage 3 or 4 chronic kidney disease (CKD) and vitamin D insufficiency.
This trial was a randomized, double-blind, placebo-controlled, multi-site study intended to establish the safety and efficacy of Rayaldee in 216 adult patients. Patients were stratified by CKD stage and randomized 2:1 to receive six months of treatment with either Rayaldee or placebo. On enrollment, all patients exhibited vitamin D insufficiency, which was corrected in 96% of patients treated with Rayaldee vs. 8% of patients treated with placebo.
Results demonstrated that all primary efficacy and safety endpoint were successfully met. The primary efficacy endpoint was a responder analysis in which “responder” was defined as any treated subject who demonstrated an average 30% decrease in plasma parathyroid hormone (PTH) from pre-treatment baseline during the last six weeks of the treatment period. A significantly higher response rate was observed with Rayaldee, which steadily increased with treatment duration. The response rate with Rayaldee was similar in CKD Stages 3 and 4.
Rayaldee is a first-in-class oral vitamin D prohormone that has a proprietary modified-release formulation designed to gradually raise serum total 25-hydroxyvitamin D (prohormone) concentrations to targeted levels (at least 30ng/mL) while avoiding upregulation of CYP24.
OPKO plans to submit a New Drug Application (NDA) to the FDA at the end of 2014.
For more information visit OPKO.com.