Canadian patients with the autoimmune liver disease primary biliary cholangitis (PBC) saw the first approval of a new drug for their condition in more than 20 years, as Health Canada approved Ocaliva (obeticholic acid).
Intercept Pharmaceuticals announced that the drug was granted marketing authorization with conditions. This type of approval — also called a Notice of Compliance with Conditions (NOC/c) — can be given to promising drugs with an acceptable safety profile while authorities await confirmatory clinical trials.
The Health Canada approval followed an accelerated priority review of the regulatory submission for Ocaliva, granted because of the unmet need for new treatments in PBC.
“We are excited to be introducing the first new treatment option for PBC in over 20 years for Canadian patients, so closely following regulatory approval in the U.S. and Europe,” Mark Pruzanski, MD, president and CEO of Intercept, said in a press release.
“Health Canada’s approval is encouraging news for patients and represents another important step in Intercept’s mission to improve the lives of people with progressive non-viral liver diseases,” he said.
The approval was granted to Ocaliva in combination with the older treatment ursodeoxycholic acid (UDCA) for adult patients who had an inadequate response to UDCA alone. The drug can also be used on its own in patients unable to tolerate UDCA.
“A substantial number of PBC patients are not achieving treatment goals with UDCA alone and a few cannot tolerate this standard of care. Until now we have only had experimental adjunctive therapies to help these non-responders with progressive disease,” said Andrew Mason, director of research for the division of gastroenterology and hepatology at the University of Alberta in Canada.
“The introduction of Ocaliva will help to address this critical need and provide an opportunity for physicians to revisit treatment goals with their patients.”
Intercept is now working to ensure both private and public health insurance plans in Canada will reimbursement Ocaliva. The company has launched the Navigate Patient Support Program to provide eligible PBC patients, who are prescribed Ocaliva by physicians, support in navigating insurance issues.
“We are very excited that Canadians living with PBC will now have an important new treatment option,” said Gail Wright, president of the Canadian PBC Society. “It is such a promising time for PBC patients, and the community has been energized by new advances in research, growing disease awareness among the public and clinicians, and now the introduction of a much-needed new therapy to help patients better manage their disease.”
Intercept is now recruiting patients for a global confirmatory Phase 3 trial (NCT02308111) of Ocaliva.