May 09, 2016 7:30am EDT
Download as PDF
Vifor Fresenius Medical Care Renal Pharma obtains rights to
commercialize RAYALDEE ® in Europe, Canada and
certain other international markets
OPKO to receive up to USD 282 million in upfront and milestone
payments, plus tiered double-digit royalties
OPKO and Vifor Fresenius Medical Care Renal Pharma to jointly
develop RAYALDEE ® for dialysis patients
ST. GALLEN, Switzerland & MIAMI--(BUSINESS WIRE)--
Vifor Fresenius Medical Care Renal Pharma (VFMCRP), a common company of
Galenica and Fresenius Medical Care, and OPKO Health (NYSE: OPK), have
entered into a collaboration and license agreement for the development
and commercialization of RAYALDEE ® in Europe, Canada, Mexico,
Australia, South Korea and certain other international markets for the
treatment of secondary hyperparathyroidism (SHPT) in patients with
chronic kidney disease (CKD) and vitamin D insufficiency. Under the
terms of the agreement, the parties will also collaborate to develop and
commercialize RAYALDEE ® for the treatment of SHPT in dialysis
patients, and OPKO has granted VFMCRP an option to acquire rights to the
US market for treatment of dialysis patients.
"Vifor Fresenius Medical Care Renal Pharma is a leader in chronic kidney
disease and we believe they are the ideal partner to bring RAYALDEE ®
to patients across Europe and other territories," said Phillip Frost,
M.D., Chairman and Chief Executive Officer of OPKO. "We believe further
developing RAYALDEE ® for dialysis patients is an important
additional step in the care of chronic kidney disease patients," Dr.
Frost continued.
“Managing bone and mineral disorders in CKD patients remains a challenge
for physicians despite the treatment options already available to them.
The clinical data for RAYALDEE ® show promising safety and
efficacy and we strongly believe that once approved, RAYALDEE ®
will become a cornerstone therapy to better address this key
co-morbidity and improve the well-being of chronic kidney disease
patients,” said Stefan Schulze, Chief Executive Officer of VFMCRP.
SHPT is a common disorder in CKD patients triggered by vitamin D
insufficiency, which can cause reduced vitamin D hormone production,
decreased intestinal absorption of dietary calcium, increased secretion
of parathyroid hormone (PTH), and metabolic bone disease. Using current
treatment options, most patients underachieve control of both vitamin D
insufficiency and SHPT, leading to a range of bone and mineral disorders.
RAYALDEE ® is an oral vitamin D prohormone treatment in a
modified release capsule being developed by OPKO. VFMCRP has entered
into an exclusive license agreement with OPKO to co-develop and
commercialize RAYALDEE ® in leading markets around the world,
including Europe (except Russia), Canada, Mexico, Australia and South
Korea. OPKO retains all rights in the US, Latin America (excluding
Mexico), Russia, China, Taiwan and Japan. The two companies will also
collaborate to prepare the Marketing Authorization Application (MAA)
submission to the European Medicines Agency (EMA).
Under the agreement, VFMCRP will make an upfront payment to OPKO of USD
50 million, plus up to an additional USD 52 million in regulatory and
launch milestones, and USD 180 million in sales-based milestones. In
addition, VFMCRP will pay OPKO tiered, double-digit royalties on sales
of the product. In the event VFMCRP exercises its option for rights to
the US dialysis market, VFMCRP will pay OPKO additional commercial-based
milestones, as well as double-digit royalties.
OPKO submitted a New Drug Application (NDA) for RAYALDEE ® to
the US Food and Drug Administration (FDA) in 2015 for the treatment of
SHPT in patients with stage 3 or 4 CKD and vitamin D insufficiency. On
29 March 2016, the FDA indicated in a Complete Response Letter (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 labelling issues with regard to RAYALDEE ® ,
nor did it request that additional studies be conducted prior to FDA
approval. OPKO subsequently resubmitted its NDA to the FDA, and the FDA
accepted OPKO’s resubmission on April 22, 2016. The new Prescription
Drug User Fee Act (PDUFA) date is October 22, 2016.
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 105 US sites.
These studies met all primary efficacy and safety endpoints, confirming
the anticipated product profile of RAYALDEE’s ability to correct vitamin
D insufficiency and treat SHPT without meaningfully increasing serum
calcium or phosphorus levels.
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 (intact parathyroid hormone).
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 US, 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.
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.
Secondary Hyperparathyroidism (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 advanced CKD.
Vifor Fresenius Medical Care Renal Pharma Ltd. , a common
company of Galenica and Fresenius Medical Care, develops and
commercialises innovative and high quality therapies to improve the life
of patients suffering from CKD worldwide. The company was founded at the
end of 2010 and is owned 55% by Galenica and 45% by Fresenius Medical
Care.
OPKO Health, Inc. is a diversified healthcare company that
seeks to establish industry-leading positions in large, rapidly growing
markets. OPKO’s diagnostics business includes BioReference Laboratories,
the third-largest clinical laboratory in the US 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. OPKO’s 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). OPKO’s 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). OPKO also has production and distribution assets worldwide,
multiple strategic investments and an active business development
strategy. More information is available at www.opko.com .
Safe Harbor Statement
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 expectations
about RAYALDEE and the success of the collaboration and licensing
agreement, whether the parties will obtain regulatory approval for,
launch or successfully commercialize RAYALDEE, whether the parties will
successfully develop RAYALDEE for the treatment of SHPT in dialysis
patients, that RAYALDEE will effectively control SHPT in patients with
stage 3 or 4 CKD by correcting vitamin D insufficiency, that RAYALDEE
will improve the well-being of CKD patients, whether payment milestones
and royalty obligations will ever be triggered, and the expected market
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
OPKO’s 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 have generated 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 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/20160509005380/en/
Vifor Fresenius Medical Care Renal Pharma
Beatrix
Benz, +41 58 851 80 16
Head of Global Communications & Public
Affairs
or
OPKO
Tara
Mackay: +1 305-575-4100
or
Media -
Terry Rooney: +1 212-223-0689
or
Marion
Janic: +1 212-223-4017
or
Investors
– LHA
Anne Marie Fields: +1 212-838-3777
or
Bruce
Voss, +1 310-691-7100
Source: OPKO Health, Inc.
Released May 9, 2016
email Email Alerts
location_city Company Profile
contact_page Contacts
rss_feed RSS News Feed
account_tree Sitemap
Apply on OPKO Health (Ireland)’s careers page