2016 Press/Media Releases:

OPKO Receives Complete Response Letter from FDA for RAYALDEE® New Drug Application

• Sole issue relates to third-party manufacturing observations
• No new clinical studies requested
• No safety or efficacy issues identified
• Management to host conference call today at 8:30 a.m. Eastern time

MIAMI (BUSINESS WIRE) March 30, 2016 – OPKO Health, Inc. (NYSE: OPK) announces that the U.S. Food and Drug Administration (FDA) has issued a complete response letter (CRL) to the Company's New Drug Application (NDA) for RAYALDEE® (calcifediol) as a treatment for secondary hyperparathyroidism (SHPT) in patients with stage 3 or 4 chronic kidney disease (CKD) and vitamin D insufficiency.

The FDA indicated in the CRL that observations of deficiencies at OPKO's third-party contract manufacturer were issued on March 25, 2016 as a result of an FDA field inspection initiated on March 14, 2016. The observations were not specific to RAYALDEE manufacturing. The CRL did not cite any safety, efficacy or labeling issues with regard to RAYALDEE, nor did it request any additional studies to be conducted prior to FDA approval.

The Company's third-party manufacturer has committed to respond promptly to the FDA's observations to ensure early resolution.

In the CRL, FDA has re-confirmed the acceptance of the proprietary name RAYALDEE. The FDA also reached agreement with OPKO for an approvable package insert and all container labeling.

"OPKO is committed to bringing RAYALDEE to patients who will benefit from its intended use and will work closely with the FDA and our third-party manufacturer to ensure that the inspection observations are promptly and fully addressed," noted Phillip Frost, M.D., Chairman and Chief Executive Officer of OPKO. "We will continue to build our commercial sales organization in preparation for the earliest possible RAYALDEE launch."

CONFERENCE CALL & WEBCAST INFORMATION

WHEN: Wednesday, March 30, 2016 at 8:30 a.m. Eastern time
DOMESTIC DIAL-IN: (866) 634-2258
INTERNATIONAL DIAL-IN: (330) 863-3454
PASSCODE: 81841862
WEBCAST: www.opko.com

For those unable to participate in the live conference call or webcast, a replay will be available beginning March 30, 2016 at 11:30 a.m. Eastern time for a period of time. To access the replay, dial (855) 859-2056 or (404) 537-3406. The replay passcode is: 81841862. The replay can also be accessed for a period of time on OPKO's website at www.opko.com.

About RAYALDEE
RAYALDEE (calcifediol) extended-release capsules are being developed for the treatment of SHPT in adult patients with stage 3 or 4 CKD and serum total 25-hydroxyvitamin D levels less than 30 ng/mL. RAYALDEE has a proprietary formulation designed to raise serum total 25-hydroxyvitamin D (prohormone) concentrations to targeted levels (at least 30 ng/mL) and to reduce elevated iPTH.

About Chronic Kidney Disease
CKD is a condition characterized by a progressive decline in kidney function. The kidney is normally responsible for excreting waste and excess water from the body, and for regulating various hormones. CKD is classified in five stages — mild (stage 1) to severe (stage 5) disease — as measured by the kidney's glomerular filtration rate.

According to the National Kidney Foundation, CKD afflicts 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 CKD. In stage 5 CKD, kidney function is minimal to absent and patients require regular dialysis or a kidney transplant for survival.

About Secondary Hyperparathyroidism (SHPT)
SHPT is a condition commonly associated with CKD in which the parathyroid glands secrete excessive amounts of parathyroid hormone (PTH). SHPT arises as a result of vitamin D insufficiency or impaired kidney function that prevents sufficient production of vitamin D hormone to properly regulate calcium and phosphorus metabolism, and PTH secretion. Prolonged elevation of blood PTH causes excessive calcium and phosphorus to be released from bone, leading to elevated serum calcium and phosphorus, softening of the bones (osteomalacia) and calcification of vascular and renal tissues. SHPT affects 40-60% of patients with moderate CKD and approximately 90% of patients with severe CKD.

About Vitamin D Insufficiency
Vitamin D insufficiency is a condition in which the body has low vitamin D stores, characterized by inadequate blood levels of vitamin D prohormone, known as 25D. An estimated 70-90% of CKD patients have vitamin D insufficiency, which can lead to SHPT and resultant debilitating bone diseases. Vitamin D insufficiency has been associated with increased mortality in CKD.

About OPKO Health, Inc.
OPKO Health, Inc. is a diversified healthcare company that seeks to establish industry-leading positions in large, rapidly growing markets. Our diagnostics business includes Bio-Reference Laboratories, the nation's third-largest clinical laboratory with a core genetic testing business and a 420-person sales force to drive growth and leverage new products, including the 4Kscore® prostate cancer test and the Claros®1 in-office immunoassay platform. Our pharmaceutical business features RAYALDEE, a treatment for SHPT in stage 3-4 CKD patients with vitamin D insufficiency, and VARUBI™ for chemotherapy-induced nausea and vomiting (oral formulation approved by FDA and launched by partner Tesaro, IV formulation in Phase 3). Our biologics business includes hGH-CTP, a once-weekly human growth hormone injection (in Phase 3 and partnered with Pfizer), and a long-acting Factor VIIa drug for hemophilia (in Phase 2a). We also have production and distribution assets worldwide, multiple strategic investments and an active business development strategy. More information is available at www.opko.com.

OPKO Health, Inc.
Investor relations
Tara Mackay
305-575-4100
- or -
Media:
Rooney & Associates
Terry Rooney
212-223-0689
- or -
Marion Janic
212-223-4017
Investors:
LHA
Anne Marie Fields
212-838-3777
or
Bruce Voss
310-691-7100

 
     
     
 
   

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