First Primary Biliary Cirrhosis Medication Submitted for Regulatory Review in Nearly Two Decades
NEW YORK, NY, USA I June 29, 2015 I Intercept Pharmaceuticals, Inc. (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.
About Primary Biliary Cirrhosis
PBC is a rare liver disease that primarily results from autoimmune destruction of the bile ducts that transport bile acids out of the liver, resulting in cholestasis. It is primarily a disease of women, afflicting approximately one in 1,000 women over the age of 40. PBC is the second leading cause of liver transplant among women in the United States. In Europe, PBC accounts for 50 percent of liver transplants due to cholestatic diseases and 6% of all liver transplants.
About Intercept
Intercept is a biopharmaceutical company focused on the development and commercialization of novel therapeutics to treat chronic, underserved liver diseases. The company’s lead product candidate, obeticholic acid (OCA), is an agonist of the farnesoid X receptor (FXR). OCA is being developed for a variety of chronic liver diseases, including primary biliary cirrhosis (PBC), nonalcoholic steatohepatitis (NASH), primary sclerosing cholangitis (PSC) and biliary atresia. The FDA has granted OCA breakthrough therapy designation for the treatment of NASH with liver fibrosis, a population representing potentially more than 14 million patients in the United States, and granted OCA fast track designation for the treatment of patients with PBC. OCA has also received orphan drug designation in both the United States and Europe for the treatment of PBC and PSC. Intercept owns worldwide rights to OCA outside of Japan, China and Korea, where it has out-licensed the product candidate to Sumitomo Dainippon Pharma. For more information about Intercept, please visit the Company’s website at: www.interceptpharma.com.
SOURCE: Intercept Pharmaceuticals
Post Views: 19
First Primary Biliary Cirrhosis Medication Submitted for Regulatory Review in Nearly Two Decades
NEW YORK, NY, USA I June 29, 2015 I Intercept Pharmaceuticals, Inc. (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.
About Primary Biliary Cirrhosis
PBC is a rare liver disease that primarily results from autoimmune destruction of the bile ducts that transport bile acids out of the liver, resulting in cholestasis. It is primarily a disease of women, afflicting approximately one in 1,000 women over the age of 40. PBC is the second leading cause of liver transplant among women in the United States. In Europe, PBC accounts for 50 percent of liver transplants due to cholestatic diseases and 6% of all liver transplants.
About Intercept
Intercept is a biopharmaceutical company focused on the development and commercialization of novel therapeutics to treat chronic, underserved liver diseases. The company’s lead product candidate, obeticholic acid (OCA), is an agonist of the farnesoid X receptor (FXR). OCA is being developed for a variety of chronic liver diseases, including primary biliary cirrhosis (PBC), nonalcoholic steatohepatitis (NASH), primary sclerosing cholangitis (PSC) and biliary atresia. The FDA has granted OCA breakthrough therapy designation for the treatment of NASH with liver fibrosis, a population representing potentially more than 14 million patients in the United States, and granted OCA fast track designation for the treatment of patients with PBC. OCA has also received orphan drug designation in both the United States and Europe for the treatment of PBC and PSC. Intercept owns worldwide rights to OCA outside of Japan, China and Korea, where it has out-licensed the product candidate to Sumitomo Dainippon Pharma. For more information about Intercept, please visit the Company’s website at: www.interceptpharma.com.
SOURCE: Intercept Pharmaceuticals
Post Views: 19
