Intercept Pharmaceuticals, Inc. (Nasdaq:ICPT) (Intercept), a
biopharmaceutical company focused on the development and
commercialization of novel therapeutics to treat progressive
non-viral liver diseases, today announced that the European
Commission has granted conditional approval for Ocaliva
(obeticholic acid) for the treatment of primary biliary cholangitis
(PBC) in combination with ursodeoxycholic acid (UDCA) in adults
with an inadequate response to UDCA or as monotherapy in adults
unable to tolerate UDCA. Ocaliva is a potent and selective agonist
of the farnesoid X receptor (FXR), which is expressed at high
levels in the liver and intestine and thought to be a key regulator
of bile acid, inflammatory, fibrotic and metabolic pathways.
“The approval of Ocaliva in Europe provides a new therapeutic
option for a substantial group of PBC patients who are not
achieving treatment goals with UDCA alone or who cannot tolerate
UDCA,” said Frederik Nevens, M.D., Ph.D., University Hospitals
Leuven & KU Leuven, Belgium, and the lead investigator of the
Phase 3 POISE clinical study. “Despite the availability of UDCA,
many patients have remained at significant risk of adverse outcomes
with no alternative treatment option available. Ocaliva can now
help fill an important unmet need for these patients.”
“We are delighted to be introducing the first new therapeutic
option for PBC in nearly 20 years in Europe where this disease is a
major reason for liver failure and a leading cause of liver
transplant in women,” said Lisa Bright, Intercept’s President,
International. “Following approval in the U.S. earlier this year,
Ocaliva’s marketing authorization in Europe represents another big
step in Intercept’s mission to provide patients with worldwide
access to our innovative therapy. This great achievement will
motivate us further to continue developing solutions that improve
the lives of people with progressive non-viral liver diseases.”
The marketing authorization allows Intercept to market Ocaliva
in 28 countries that are member states of the European Union, as
well as 3 additional European Economic Area member states. As
conditions of the approval, Intercept is required to provide
post-approval updates on safety and efficacy analyses for Ocaliva
from the ongoing Phase 4 COBALT outcomes study and a short-term
study in patients with hepatic impairment.
“As a community, our priority is to advocate for changes which
ensure that people diagnosed with PBC have the best possible
prognosis,” said Tatjana Reic, President of the European Liver
Patients Association (ELPA). “With this in mind, we are excited
about this advance for patients with an inadequate response or
intolerability to the current available treatment. Such patients
will soon have access to a new treatment option to manage their
PBC.”
The marketing authorization was based on efficacy and safety
data derived from three randomized double-blind, placebo-controlled
clinical trials evaluating the effect of Ocaliva on alkaline
phosphatase (ALP) and bilirubin in patients with PBC. It was also
supported by two clinical databases that include more than 10,000
patients from the Global PBC Study Group and UK-PBC Group, both
independently confirming that achieving lower ALP and/or bilirubin
levels is significantly correlated with increased transplant-free
survival.
In the Phase 3 POISE study, nearly half of patients (46%) in the
titration group treated with Ocaliva in combination with UDCA
achieved the primary endpoint compared to 10% in the control group
(placebo added to UDCA) (p<0.0001). Additionally, 77% of
patients taking Ocaliva in combination with UDCA achieved a
reduction of more than 15% in ALP at 12 months, compared to 29%
taking UDCA alone.
The most commonly reported adverse reactions were pruritus (63%)
and fatigue (22%). Adverse reactions leading to discontinuation
were 1% in the Ocaliva titration arm and 11% in the Ocaliva 10 mg
arm. The most common adverse reaction leading to discontinuation
was pruritus. The majority of pruritus occurred within the first
month of treatment and tended to resolve over time with continued
dosing.
About Primary Biliary Cholangitis Primary
biliary cholangitis (PBC) is a rare, autoimmune cholestatic liver
disease that puts patients at risk for life-threatening
complications. PBC is primarily a disease of women, afflicting
approximately one in 1,000 women over the age of 40. If left
untreated, survival of PBC patients is significantly worse than the
general population.
About Ocaliva® (obeticholic acid)Ocaliva
(obeticholic acid) is a potent and highly selective agonist of the
farnesoid X receptor (FXR), a nuclear receptor expressed in the
liver and intestine. FXR is a key regulator of bile acid,
inflammatory, fibrotic and metabolic pathways.
In December 2016, Ocaliva received conditional marketing
authorization in Europe for the treatment of PBC in combination
with ursodeoxycholic acid (UDCA) in adults with an inadequate
response to UDCA or as monotherapy in adults unable to tolerate
UDCA, conditional to the company providing further data
post-approval to confirm benefit. In May 2016, the U.S. Food and
Drug Administration granted accelerated approval to Ocaliva for the
treatment of PBC. For full prescribing information in the U.S.,
visit Ocaliva.com.
EU IMPORTANT SAFETY INFORMATION
ContraindicationsHypersensitivity to the active
substance or to any of the excipients and complete biliary
obstruction.
Warnings and PrecautionsElevations in alanine
amino transferase (ALT) and aspartate aminotransferase (AST) have
been observed in patients taking obeticholic acid. Clinical signs
and symptoms of hepatic decompensation have also been observed.
These events have occurred as early as within the first month of
treatment. Liver-related adverse events have primarily been
observed at doses higher than the maximum recommended dose of 10 mg
once daily. Patients should be monitored during treatment with
Ocaliva for elevations in liver biochemical tests and for the
development of liver-related adverse events. Dosage adjustments are
needed for patients with moderate (Child-Pugh Class B) or severe
(Child-Pugh Class C) hepatic impairment.
Severe pruritus was reported in 23% of patients treated with
Ocaliva 10 mg arm, 19% of patients in the Ocaliva titration arm and
7% of patients in the placebo arms. The median time to onset of
severe pruritus was 11, 158 and 75 days for patients in the Ocaliva
10 mg, Ocaliva titration and placebo arms, respectively. Management
strategies include the addition of bile acid binding resins or
antihistamines, dose reduction, reduced dosing frequency and/or
temporary dose interruption.
Adverse ReactionsThe most commonly reported
adverse reactions were pruritus (63%) and fatigue (22%). Other
common adverse reactions observed in clinical trials (> 5%) were
abdominal pain and discomfort, rash, oropharyngeal pain, dizziness,
constipation, arthralgia, thyroid function abnormality and
eczema.
Drug InteractionBile acid binding resins such
as cholestyramine, colestipol or colesevelam adsorb and reduce bile
acid absorption and may reduce efficacy of obeticholic acid.
When concomitant bile acid binding resins are administered,
obeticholic acid should be taken at least 4-6 hours before or 4-6
hours after taking a bile acid binding resin, or at as great
an interval as possible.
For detailed safety information for Ocaliva (obeticholic acid) 5
mg and 10 mg tablets including posology and method of
administration, special warnings, drug interactions and adverse
drug reactions, please see the European Summary of Product
Characteristics that can be found on www.ema.europa.eu once
posted.
About the POISE StudyThe POISE trial studied
the safety and efficacy of once-daily treatment with Ocaliva in PBC
patients with an inadequate therapeutic response to, or who are
unable to tolerate, UDCA, the current standard of care. Of 216
patients randomized to three treatment arms—placebo, Ocaliva 5 mg
titrated to 10 mg or Ocaliva 10 mg—93% continued receiving UDCA.
The Ocaliva 5-10 mg titration group received Ocaliva 5 mg for six
months, after which dosing was increased to 10 mg based on
tolerability and biochemical response. The study's primary endpoint
was a reduction in ALP to below a threshold of 1.67 times the upper
limit of normal, with a minimum of 15% reduction in ALP level from
baseline, and a normal bilirubin level after 12 months of
therapy.
About InterceptIntercept is a biopharmaceutical
company focused on the development and commercialization of novel
therapeutics to treat progressive non-viral liver diseases,
including primary biliary cholangitis (PBC), nonalcoholic
steatohepatitis (NASH), primary sclerosing cholangitis (PSC) and
biliary atresia. Founded in 2002 in New York, Intercept now has
operations in the United States, Europe and Canada. For more
information about Intercept, please visit
www.interceptpharma.com.
Safe Harbor Statements This press release
contains "forward-looking statements" within the meaning of the
Private Securities Litigation Reform Act of 1995, including, but
not limited to, statements regarding the clinical relevance and
utility of ALP and the surrogate endpoint used in the Phase 3 POISE
trial to predict clinical outcomes, the acceptance of Ocaliva®
(obeticholic acid) as a treatment for PBC by healthcare providers,
patients and payors, the potential approval of OCA in PBC by
regulatory bodies outside the United States and Europe and the
timelines related thereto, the availability of OCA for the
treatment of PBC other jurisdictions outside the United States and
Europe and timelines related thereto, the anticipated prevalence of
and other epidemiological estimates and market data related to PBC,
the continued development of OCA and Intercept's other product
candidates, and our strategic directives under the caption "About
Intercept." These "forward-looking statements" are based on
management's current expectations of future events and are subject
to a number of important risks and uncertainties that could cause
actual results to differ materially and adversely from those set
forth in or implied by such forward-looking statements. These risks
and uncertainties include, but are not limited to: Intercept's
ability to successfully commercialize Ocaliva in PBC, and
Intercept's ability to maintain its regulatory approval of Ocaliva
in the United States for Ocaliva in PBC; the initiation, cost,
timing, progress and results of Intercept's development activities,
preclinical studies and clinical trials, including Intercept’s
development program in NASH; the timing of and Intercept's ability
to obtain and maintain regulatory approval of OCA in PBC in
countries outside the United States and in indications other than
PBC and any other product candidates it may develop such as
INT-767; conditions that may be imposed by regulatory authorities
on Intercept's marketing approvals for its product candidates such
as the need for clinical outcomes data (and not just results based
on achievement of a surrogate endpoint), and any related
restrictions, limitations, and/or warnings in the label of any
approved product candidates; Intercept's plans to research, develop
and commercialize its product candidates; Intercept's ability to
obtain and maintain intellectual property protection for its
product candidates; Intercept's ability to successfully
commercialize OCA in indications other than PBC and its other
product candidates; the size and growth of the markets for
Intercept's product candidates and its ability to serve those
markets; the rate and degree of market acceptance of any of
Intercept's products, which may be affected by the reimbursement
that it may receive for its products from payors; the success of
competing drugs that are or become available; the election by
Intercept's collaborators to pursue research, development and
commercialization activities; Intercept's ability to attract
collaborators with development, regulatory and commercialization
expertise; regulatory developments in the United States and other
countries; the performance of third-party suppliers and
manufacturers; Intercept's need for and ability to obtain
additional financing; Intercept's estimates regarding expenses,
future revenues and capital requirements and the accuracy thereof;
Intercept's use of cash, short-term investments and the proceeds
from the offering; Intercept's ability to attract and retain key
scientific or management personnel; and other factors discussed
under the heading "Risk Factors" contained in our annual report on
Form 10-K for the year ended December 31, 2015 filed on February
29, 2016 as well as any updates to these risk factors filed from
time to time in our other filings with the Securities and Exchange
Commission. All information in this press release is as of the date
of the release, and Intercept undertakes no duty to update this
information unless required by law.
Contact
For more information about Intercept Pharmaceuticals, please contact:
Mark Vignola
+1-646-747-1000
investors@interceptpharma.com
Christopher Frates
+1-646-757-2371
media@interceptpharma.com
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