OPKO's Second Rayaldee Phase 3 Study Achieves Primary Endpoints - Quick Facts

(RTTNews) - OPKO Health, Inc. (OPK) announced top-line results from the second and final pivotal phase 3 trial of Rayaldee. This trial is the second of two identical randomized, double-blind, placebo-controlled, multi-site studies planned to establish the safety and efficacy of Rayaldee as a new treatment for secondary hyperparathyroidism or SHPT in patients with stage 3 or 4 chronic kidney disease or CKD and vitamin D insufficiency. Both studies are the subject of a Special Protocol Assessment established with the U.S. Food and Drug Administration or FDA in August 2012.

"Top-line data from this second study confirmed that Rayaldee effectively controls secondary hyperparathyroidism in patients with stage 3 or 4 chronic kidney disease by correcting vitamin D insufficiency," stated Joel Z. Melnick, M.D., Vice President of Clinical Research and Development for OPKO's Renal Division. "As with the first trial, Rayaldee was equally effective in both disease stages, indicating that this new therapy is appropriate even for patients with minimal functioning kidney mass."

The study involved 216 adult patients recruited from 38 sites throughout the U.S. Patients were stratified by CKD stage and randomized in a 2:1 fashion to receive 6 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.

A New Drug Application submission to the FDA is planned for the end of 2014.