First new medication for primary biliary
cholangitis in nearly 20 years
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
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