March 30, 2016 2:00am EDT
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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)--
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
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 .
This press release contains “forward-looking statements,” as that
term is defined under the Private Securities Litigation Reform Act of
1995 (PSLRA), regarding product development efforts and other
non-historical facts about our expectations, beliefs or intentions
regarding our business, technologies and products, financial condition,
strategies or prospects, including statements regarding RAYALDEE, our
ability to obtain regulatory approval for and launch RAYALDEE,
expectations about RAYALDEE, that RAYALDEE will effectively control
secondary hyperparathyroidism in patients with stage 3 or 4 chronic
kidney disease by correcting vitamin D insufficiency, the expected
market potential for RAYALDEE, whether adequate responses to the FDA
observations will be timely submitted for early resolution, whether we
will successfully build out our commercial sales organization, and our
expected approval and launch dates for RAYALDEE. Many factors could
cause our actual activities or results to differ materially from the
activities and results anticipated in forward-looking statements. These
factors include those described in our filings with the Securities and
Exchange Commission, as well as risks inherent in funding, developing
and obtaining regulatory approvals of new, commercially-viable and
competitive products and treatments, including the risks that our
third-party manufacturer may not be able to resolve the deficiencies
noted by the FDA in a timely matter or at all, that the FDA could
identify additional issues that affect our ability to obtain regulatory
approval, that the phase 3 clinical trials for RAYALDEE may not have
generated data that would support the approval or marketing of this
product for the indications being pursued, that others may develop
products which are superior to RAYALDEE, and that RAYALDEE may not have
advantages or prove to be superior over presently marketed products,
including the currently used high monthly doses of prescription vitamin D 2 ,
activated vitamin D hormone and over-the-counter vitamin D supplements.
In addition, forward-looking statements may also be adversely affected
by general market factors, competitive product development, product
availability, federal and state regulations and legislation, the
regulatory process for new products and indications, manufacturing
issues that may arise, patent positions and litigation, among other
factors. The forward-looking statements contained in this press release
speak only as of the date the statements were made and we do not
undertake any obligation to update forward-looking statements. We intend
that all forward-looking statements be subject to the safe-harbor
provisions of the PSLRA.
View source version on businesswire.com: http://www.businesswire.com/news/home/20160329006635/en/
Investor relations, Tara Mackay
or
Rooney
& Associates
Terry Rooney, 212-223-0689
or
Marion
Janic, 212-223-4017
or
LHA
Anne
Marie Fields, 212-838-3777
or
Bruce
Voss, 310-691-7100
Source: OPKO Health, Inc.
Released March 30, 2016
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