FDA Accepts OPKO’s New Drug Application for Rayaldee™
July 28 2015 - 01:41PM
Business Wire
OPKO Health, Inc. (NYSE: OPK) today announced that the U.S. Food
and Drug Administration (FDA) has accepted for review OPKO’s New
Drug Application (NDA) for calcifediol modified-release capsules
(proposed trade name, Rayaldee™). In the NDA, OPKO seeks approval
of Rayaldee for the prevention and treatment of secondary
hyperparathyroidism (SHPT) in patients with stage 3 or 4 chronic
kidney disease (CKD) and vitamin D insufficiency.
“FDA acceptance of this NDA should be cheered by healthcare
professionals who care for CKD patients,” commented Dr. Kevin J.
Martin, Director of the Division of Nephrology at Saint Louis
University School of Medicine. “If approved, Rayaldee will provide
a new therapeutic option for controlling elevated parathyroid
hormone levels in this large and undertreated patient
population.”
OPKO expects written notification of NDA acceptance along with
the Prescription Drug User Fee Act (PDUFA) date in the FDA’s 74-Day
letter, which the Company expects to receive by mid-August
2015.
The NDA is supported by data from three randomized,
double-blind, placebo-controlled studies and one open-label
extension study conducted in the targeted patient population at a
total of 105 U.S. sites. These studies met all primary efficacy and
safety endpoints, as previously announced.
About Rayaldee
Rayaldee (calcifediol modified-release capsules) is an oral
vitamin D prohormone treatment being developed for SHPT in patients
with stage 3 or 4 CKD and vitamin D insufficiency. It has a
proprietary modified-release formulation designed to gradually and
reliably raise serum total 25-hydroxyvitamin D (prohormone)
concentrations to targeted levels (at least 30 ng/mL) while
avoiding upregulation of CYP24A1, a cytochrome P-450 enzyme which
interferes with the desired parathyroid hormone (PTH)-lowering
effect. Gradual elevation of serum total 25-hydroxyvitamin D is
intended to prevent excessive elevation of serum calcium and
related vascular and renal calcification.
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 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 25-hydroxyvitamin D. 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 Secondary Hyperparathyroidism (SHPT)
SHPT is a condition commonly associated with CKD in which the
parathyroid glands secrete excessive amounts of 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 OPKO
OPKO is a multinational biopharmaceutical and diagnostics
company that seeks to establish industry leading positions in
large, rapidly growing markets by leveraging its discovery,
development and commercialization expertise and novel and
proprietary technologies. For more information, visit
http://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 the expected receipt of the 74-Day letter from the FDA,
the contents of the 74-Day Letter and our ability to address the
same, the expected PDUFA date and whether Rayaldee will be approved
by the FDA, our ability to successfully launch and commercialize
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, whether Rayaldee is appropriate for patients with
minimal functioning kidney mass and its efficacy during longer term
administration, whether Rayaldee will be highly effective in
correcting vitamin D insufficiency, allowing more reliable
treatment of patients, market potential for Rayaldee, and that we
will be able to successfully develop, obtain approval for and
launch sales of 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 the phase 3 clinical trials for Rayaldee
may not generate data that would support the approval or marketing
of this product for the indications being studied, 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 D2, 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/20150728006530/en/
OPKO Health, Inc.Charles W. Bishop, PhD, 305-575-4100CEO,
Renal DivisionorMedia Contacts:Rooney & AssociatesTerry
Rooney, 212-223-0689trooney@rooneyco.comorRooney &
AssociatesMarion Janic, 212-223-4017mjanic@rooneyco.com
Opko Health (NASDAQ:OPK)
Historical Stock Chart
From Feb 2024 to Mar 2024
Opko Health (NASDAQ:OPK)
Historical Stock Chart
From Mar 2023 to Mar 2024