First new
medication for primary biliary cholangitis in nearly 20
years
Rapid NICE
approval only two months after marketing authorization in the EU;
one of the fastest approvals to date for an orphan
medicine
NEW YORK, March 02, 2017 (GLOBE
NEWSWIRE) -- Intercept Pharmaceuticals, Inc. (Nasdaq:ICPT)
(Intercept) today announced that the National Institute for Health
and Care Excellence (NICE) has approved Ocaliva (obeticholic acid)
for routine use by the National Health Service (NHS) in England,
Wales and Northern Ireland. Ocaliva has been conditionally approved
in the European Union 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. The NHS is expected to make Ocaliva
available to patients with PBC within 90 days of NICE's final
appraisal publication and Intercept will work with local
reimbursement authorities to help ensure eligible patients obtain
access.
Although it is a rare disease, PBC is a leading
cause of liver transplantation in adult women in the UK. Ocaliva is
a new treatment option for patients with PBC who do not fully
respond to, or are intolerant to, current treatment and remain at
risk of their disease progressing toward cirrhosis, liver
transplantation or death.
"I am excited to see that the substantial group of
PBC patients who are not achieving treatment goals with UDCA alone
or who cannot tolerate UDCA will soon be able to access the first
new therapeutic option in nearly 20 years. This truly is good news
for our PBC patients," said David Jones, M.D., Ph.D., Professor of
Liver Immunology at Newcastle University and Consultant
Hepatologist at Newcastle upon Tyne Hospitals Trust, which hosts
one of Europe's leading clinical services in the disease. "The
development of new treatments for PBC is a powerful example of the
medical innovation that can occur when government, industry,
academia, community clinicians and, most importantly, patients come
together to address an unmet need."
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. In December
2016, Ocaliva received conditional marketing authorization in
Europe 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. The marketing authorization 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.
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.
"This very rapid decision by NICE, one of the
fastest approvals to date for an orphan medication, is an important
affirmation of the scientific innovation, clinical value and
cost-effectiveness of Ocaliva by one of the most respected health
technology assessment bodies," said Lisa Bright, Intercept's
President, International. "We welcome NICE's decision to provide
broad access to Ocaliva and we owe a tremendous debt to people
living with PBC and the clinical groups who helped us to achieve
this milestone for the PBC community."
"It is exciting news for PBC patients that this
new treatment option will now be routinely available in England,
Wales and Northern Ireland," said Collette Thain MBE, CEO of The
PBC Foundation. "When I was diagnosed with PBC, UDCA was the only
approved treatment option and PBC wasn't a major priority for many
researchers. Thankfully, so much has changed for people living with
PBC since then. After decades of advocacy from the PBC community,
we have a new treatment option, a growing awareness of the disease
among the general public, greater expertise amongst clinicians and
an acceleration of PBC research in the UK and around the
globe."
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.
PBC in the UK
The estimated prevalence of PBC in the UK is
approximately 3.9 per 10,000 population, equating to approximately
19,175 people in England.
Patients and medical leaders in the UK have played
a critical role in accelerating PBC research, innovation and
awareness globally. The patient community in the UK has
guided Intercept's efforts to improve PBC education and understand
the unmet needs of patients and their families. The UK-PBC Study
Group, a research consortium funded by the UK government through
the Medical Research Council and National Institute for Health
Research, played a critical role in the development of Ocaliva and
was one of two clinical databases to independently confirm that
achieving lower ALP and/or bilirubin levels is significantly
correlated with increased transplant-free survival.
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
Contraindications
Hypersensitivity to the active substance or to any
of the excipients and complete biliary obstruction.
Warnings and
Precautions
Elevations 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
Reactions
The 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
Interaction
Bile 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.
About
Intercept
Intercept 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. Intercept's International headquarters
are located in London. 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, bilirubin 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 commercial availability of OCA for the treatment of PBC
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 in
jurisdictions in which Ocaliva is approved for use 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 ones in which it is approved
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
products and 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 products and 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 products and
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 products and 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, 2016 filed on
March 1, 2017 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
This
announcement is distributed by Nasdaq Corporate Solutions on behalf
of Nasdaq Corporate Solutions clients.
The issuer of this announcement warrants that they are solely
responsible for the content, accuracy and originality of the
information contained therein.
Source: Intercept Pharmaceuticals, Inc. via
Globenewswire
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