OPKO's second Rayaldee Ph III trial meets primary endpoints

US drugmaker OPKO Health (NYSE: OPK) has released successful top-line results from the second and final pivotal Phase III trial of Rayaldee, a first-in-class oral vitamin D prohormone treatment.

This trial is the second of two identical randomized, double-blind, placebo-controlled, multi-site studies intended to establish the safety and efficacy of Rayaldee as a new treatment for secondary hyperparathyroidism (SHPT) in patients with stage 3 or 4 chronic kidney disease (CKD) and vitamin D insufficiency. Both trials are the subject of a Special Protocol Assessment established with the US Food and Drug Administration in August 2012. A New Drug Application submission to the FDA is planned for the end of 2014.

“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 Melnick, vice president of clinical R&D for OPKO’s Renal Division, adding: “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.”