Global Liver Institute Frustrated by FDA GIDAC Committee Failure to Recognize Serious, Unmet Need of Liver Patients in Rejection of Obeticholic Acid for Pre-Cirrhotic Fibrosis Due to NASH
May 22, 2023, Washington, DC – On Friday, May 19, the Food and Drug Administration’s Gastrointestinal Drugs Advisory Committee (GIDAC) voted to reject Intercept Pharmaceutical’s application for obeticholic acid (OCA) as a treatment for NASH. Despite acknowledging OCA met the endpoint established by the FDA of reducing fibrosis by at least one stage, GIDAC voted against approval for treatment for NASH. After this morning’s Intercept Pharmaceuticals investor call, our hope is that Intercept and other drug developers persist in their investments in non-viral liver diseases despite this setback.
The FDA’s GIDAC has failed to recognize the serious, unmet need of liver patients with NASH. By guessing hypothetical consequences of drug-induced liver injury against real-world patient impact without therapy, GIDAC has usurped patient/provider decision-making of benefits and individual risk. GIDAC’s fear of scaling risk ignores the real risks to current patients every day.
As Intercept met the endpoint designated by the FDA, Agency staff should not argue about the validity of that endpoint during the Advisory Committee. This unfairly shifts the regulatory goal after the fact. Actual experts and the patient community were unified in testifying that medication is needed now, not in 2-3 years when the outcome trial is finished. As Donna R. Cryer, JD, President and CEO of Global Liver Institute, stated in her testimony “Unlike Pruritus and elevated LDL, death is not a manageable side effect.”
As the authority for approval, patients rely on the FDA for a consistent, evidence-based regulatory process that prioritizes the patient experience. Patients with chronic diseases need a reliable regulatory partner to address life-impacting and life-threatening conditions. To address this sufficiently, the FDA should consider reorganizing its Committees with the realignment of their Divisions to better determine helpful patient outcomes.
With this vote, NASH patients are again left without a therapy to slow their chronic, progressive disease. Drugs with a higher efficacy and better safety profile should be approved, but we are concerned that patients do not have a regulatory partner who is acting in good faith.
About Global Liver Institute
Global Liver Institute (GLI) was built to solve the problems that matter to liver patients, equipping advocates to improve the lives of individuals and families impacted by liver disease. GLI promotes innovation, encourages collaboration, and supports the scaling of optimal approaches to help eradicate liver diseases. GLI believes liver health must take its place on the global public health agenda commensurate with the prevalence and impact of liver illness. GLI is the only patient-created, patient-driven nonprofit organization tackling liver health and all liver disease holistically, operating globally. Follow GLI on Facebook, Instagram, LinkedIn, and YouTube.