The National Organization for Rare Disorders (NORD) gave its 2017 Industry Innovation Award to Intercept Pharmaceuticals for the company’s primary biliary cholangitis (PBC) drug Ocaliva (obeticholic acid).
PBC, a rare, autoimmune cholestatic liver disease, affects one in 1,000 women over 40 years old. If left untreated, it can be life-threatening. Intercept received the award because of its commitment to the rare disease community and the impact Ocaliva has had on the lives of PBC patients.
“We are honored to be a recipient of the 2017 Industry Innovation Award for the development of Ocaliva, the first medication approved for patients with PBC in nearly 20 years,” Dr. David Shapiro, Intercept’s chief medical officer, said in a press release. “We’d like to thank the PBC community for their tremendous efforts supporting innovative research and for being an invaluable partner to Intercept as we have worked to bring Ocaliva to patients in need.”
In May 2016, Ocaliva received accelerated approval from the U.S. Food and Drug Administration (FDA) – in combination with ursodeoxycholic acid (UDCA) — to treat adults with PBC who respond inadequately to UDCA, or as monotherapy in adults who cannot tolerate UDCA. Continued approval might be contingent upon verification and description of clinical benefit in confirmatory trials.
In December 2016, the European Union followed suit, giving Ocaliva conditional marketing authorization for the same indication. Intercept is now assessing Ocaliva’s potential to treat primary sclerosing cholangitis and biliary atresia, two other rare liver diseases that lack approved medications.
“We are proud to present the team at Intercept with a 2017 Industry Innovation Award for the important work they have done to address the serious unmet need in PBC,” added NORD’s president and CEO, Peter L. Saltonstall. “We hope the research programs evaluating obeticholic acid for the treatment of patients with other rare liver diseases lead to further advances for the rare disease community.”
Intercept is now enrolling patients in its Phase 3 COBALT study (NCT02308111), investigating Ocaliva in patients with primary biliary cirrhosis, aiming to confirm clinical benefit for regulatory purposes. For more information on enrollment criteria and the 159 trial locations, visit the trial page or contact study director David Shapiro directly.