Opko Health Rockets on FDA Approval of Rayaldee Four Months Ahead of PDUFA Date

June 21, 2016
By Alex Keown, BioSpace.com Breaking News Staff

MIAMI – Shares of Opko Health jumped nearly 5 percent this morning after the U.S. Food and Drug Administration (FDA) approved Opko Health’s Rayaldee, extended release capsules for the treatment of secondary hyperparathyroidism (SHPT) in adults with stage 3 or 4 chronic kidney disease.

The approval comes four months ahead of what Opko initially anticipated after it filed a resubmitted New Drug Application to the FDA in April. Initially, the company anticipated the FDA to make its decision in October.

The FDA’s approval comes after the completion of two 26-week phase III trials which demonstrated that Vitamin D insufficiency was corrected in more than 80 percent of the patients receiving Rayaldee compared with less than 7 percent of subjects receiving placebo. Rayaldee is an extended release product containing 30 mcg of a prohormone called calcifediol, which is used to determine the level of a patient’s vitamin D status. Rayaldee is the first drug approved for SHPT in chronic kidney disease, Opko said in a statement.

"Rayaldee fills a large void in the current treatment options for SHPT in predialysis patients," Charles Bishop, chief executive officer of OPKO's Renal Division, said in a statement. "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."

Chronic kidney disease (CKD) affects over 26 million people in the U.S., including more than 20 million patients with moderate (stages 3 or 4) and severe (stage 5) forms of the condition. An estimated 70 to 90 percent of CKD patients have vitamin D insufficiency, which can lead to secondary hyperparathyroidism (SHPT). It is estimated that SHPT affects 40 to 60 percent of patients with moderate CKD and approximately 90 percent of patients with severe CKD.

In March, the FDA sent a Complete Response Letter for Rayaldee due to issues with the third-party manufacturer the company was using at the time. However, in April the FDA accepted Opko’s resubmitted New Drug Application for Rayaldee, with regulators set to make a decision on the drug in October. The FDA did not force the company to redo any clinical trials.

Hailing the U.S. approval of Rayaldee as a milestone for Opko, saying the drug is “one of Opko’s many pharmaceutical products being developed for significant medical problems which will benefit from new treatment options." Opko expects to launch Rayaldee in the U.S. later this year.

In May, Opko which forged a $282 million deal with Swiss-based Vifor Fresenius Medical Care Renal Pharma to commercialize Rayaldee in Europe, Canada, Mexico, Australia, South Korea and certain other international markets.