Biotechnology Events

Home

Intercept Pharmaceuticals, Inc.

.

PLEASE SCROLL DOWN TO THE RESULTS SECTION (below) TO SEE THE OUTCOME OF THIS EVENT.

.

Mark Pruzanski, M.D., CEO & President, commented on the MAA for OCA. He stated, "For our European marketing application, our regulatory review continues. We anticipate marketing approval near year-end with the European launch thereafter. As such, we do not anticipate ex-U.S. OCA revenues until 2017."
Source: Q4 2015 earnings conference call, 2/22/16.

.

Intercept Pharmaceuticals Submits Applications in the U.S. and Europe for Marketing Approval of Obeticholic Acid for the Treatment of Primary Biliary Cirrhosis

- First Primary Biliary Cirrhosis Medication Submitted for Regulatory Review in Nearly Two Decades -

NEW YORK, June 29, 2015 (GLOBE NEWSWIRE) -- Intercept Pharmaceuticals, Inc. (Nasdaq:ICPT) (Intercept), a clinical stage biopharmaceutical company focused on the development and commercialization of novel therapeutics to treat chronic underserved liver diseases, today announced the achievement of two important regulatory milestones for obeticholic acid (OCA) in primary biliary cirrhosis (PBC): submission of a New Drug Application (NDA) for accelerated approval to the U.S. Food and Drug Administration (FDA) and acceptance of the Marketing Authorization Application (MAA) by the European Medicines Agency (EMA). Intercept is seeking approval of OCA 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.

UDCA is currently the only approved medication for the treatment of PBC and is the standard of care for all PBC patients. However, a majority of patients continue to experience persistent elevations above the upper limit of normal in the serum marker alkaline phosphatase (ALP), corresponding with increased risk of liver failure, need for liver transplant and death. Thus, there continues to be a critical need for new treatments for patients with PBC.

The NDA and MAA submissions include a total of 1,507 subjects exposed to at least a single dose of OCA. Of these subjects, 432 were patients with PBC, with 290 treated for at least six months and 232 treated for at least one year. The key efficacy and safety data are derived from three randomized double-blind, placebo-controlled clinical trials in patients with PBC evaluating the effect of OCA on ALP and bilirubin. All three trials met their primary endpoints with high statistical significance and improvements were seen in secondary endpoints including markers of liver injury, immunity, inflammation and apoptosis. OCA treatment was generally well-tolerated, with primarily mild or moderate pruritus being the most common adverse event.

The regulatory submissions are 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.

"Over the past several years, the medical community has gained a deeper appreciation of the extent of the unmet medical need in PBC," said Mark Pruzanski, M.D., President and Chief Executive Officer. "In each of our PBC clinical trials, OCA has demonstrated the ability to rapidly and sustainably lower ALP and improve bilirubin levels, both when added to UDCA and as monotherapy. If approved, we believe OCA will become an important new treatment option that will help patients with PBC."

OCA has received orphan drug designation for PBC in both the United States and Europe and fast track designation for PBC in the United States. In December 2014, Intercept initiated its rolling NDA submission for accelerated approval of OCA in PBC and began a Phase 3b confirmatory clinical outcomes trial in PBC, in accordance with FDA accelerated approval regulations. This trial is anticipated to enroll approximately 350 patients with advanced PBC at 150 centers in more than 20 countries, and is expected to be completed on a post-marketing basis.
More
Source: press release, 6/29/15. http://ir.interceptpharma.com/releasedetail.cfm?ReleaseID=919926

.

Compound/DeviceSpecialtyIndicationCompound ClassTarget
Obeticholic acid (OCA) PBCGastroenterologyPrimary Biliary Cirrhosis (PBC)Farnesoid X Receptor (FXR) AgonistFarnesoid X Receptor (FXR)

Mechanism of action: Obeticholic acid (OCA) is a bile acid analog and first-in-class Farnesoid X Receptor (FXR) agonist derived from the primary human bile acid chenodeoxycholic acid, or CDCA.

Phase of Development: Filed

Event Type: Regulatory EMA: CHMP Opinion (Estimate)

Dates: 2016-09-16

Results:

.

European Commission Grants Intercept's Ocaliva® (obeticholic acid) Marketing Authorization for the Treatment of Primary Biliary Cholangitis

NEW YORK, Dec. 14, 2016 (GLOBE NEWSWIRE) -- 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.
More
Source: press release, 12/14/16. http://ir.interceptpharma.com/releasedetail.cfm?ReleaseID=1004114

.

Intercept Pharmaceuticals Receives Positive CHMP Opinion for Ocaliva® (Obeticholic Acid) for the Treatment of Primary Biliary Cholangitis in the European Union

NEW YORK, Oct. 14, 2016 (GLOBE NEWSWIRE) -- Intercept Pharmaceuticals, Inc. (Nasdaq:ICPT) (Intercept), a biopharmaceutical company focused on the development and commercialization of novel therapeutics to treat non-viral, progressive liver diseases, today announced that the European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion recommending marketing authorization of the Company's Marketing Authorization Application (MAA) for obeticholic acid (OCA), an FXR agonist, for the treatment of primary biliary cholangitis (PBC) conditional to the company providing further data post-approval to confirm benefit.

Ursodeoxycholic acid (UDCA) is currently the only approved medication for the treatment of PBC in Europe and is the standard of care for all PBC patients. However, a substantial percentage of patients treated with UDCA continue to experience persistent elevations above the upper limit of normal in the serum marker alkaline phosphatase (ALP), which has been shown to correspond with increased risk of liver failure, need for liver transplant and death. Patients with PBC also face a risk of experiencing adverse outcomes when bilirubin levels are elevated. Total bilirubin levels, even within the normal range, have been shown to predict clinical outcomes in PBC.

"Although it is a rare disease, PBC remains one of the most common indications for liver transplant among women in Europe," 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. "There is substantial unmet need in this disease and a real urgency around the need for new therapies to help the many PBC patients who are either intolerant of the single existing approved therapy ursodeoxycholic acid or don't respond to it sufficiently to protect their livers and prevent the development of cirrhosis and the need for transplant."

The MAA submission included data from more than 1,500 subjects exposed to at least a single dose of OCA. The positive opinion of the CHMP was based on efficacy and safety data derived from three randomized double-blind, placebo-controlled clinical trials in patients with PBC evaluating the effect of OCA on ALP and bilirubin. The MAA submission 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 CHMP opinion will form the basis for a European Commission (EC) decision as to whether to formally grant the conditional marketing authorization for OCA with unified labelling in the 28 countries that are Member States of the European Union, as well as European Economic Area members Iceland, Liechtenstein and Norway. As the conditions for approval, Intercept is required to provide post-approval updates on safety and efficacy analyses for OCA from the ongoing COBALT outcomes trial and a short-term trial in patients with hepatic impairment.

"We owe a tremendous debt to the many patients and physicians whose participation in the research program for OCA led to this positive outcome," said Lisa Bright, Intercept's President, International. "In addition to playing a critical role in the development of OCA, the PBC community in Europe has been the driving force in establishing the two major patient databases that have been so central to recent advances in our understanding of the disease. There is a palpable sense of excitement about the growth of PBC awareness in Europe, and the CHMP's positive opinion on OCA brings us one step closer to introducing the first new therapy for PBC in approximately two decades."
More
Source: press release, 10/14/16. http://ir.interceptpharma.com/releasedetail.cfm?ReleaseID=993583

.