FDA has scheduled an adcom (advisory committee meeting) for Intercept's candidate drug to be the first approved treatment for NASH.

This is the public's opportunity to provide input to FDA as part of its deliberations about approving a drug. If you would like to have a chance to add your views to the discussion, this is how you do it.

Key information for patients & caregivers:

• When the Food and Drug Administration (FDA) is considering approving a new product, it often seeks the opinions of an advisory committee – an independent group of experts including physicians, statisticians, researchers and others – through a process called an advisory committee meeting.
• In the case of new drug applications (NDAs), the purpose of the meeting is to help the committee understand the burden of disease and how the product may be received in the real world.
• Intercept is anticipating that the FDA will hold an advisory committee meeting for their NDA for OCA in pre-cirrhotic liver fibrosis due to NASH later this spring, May 19,2023.
• The voices of patients, caregivers, and patient advocacy groups are an important part of the meeting and there are two ways to participate: speaking during the open public hearing (OPH) or submitting a letter of support ahead of the meeting. Both are rare opportunities to share your insights and help the advisory panel shape their evaluation.
• The advisory committee meeting is usually a full day (virtual), however, the OPH is a dedicated ~1 hour in the afternoon, and individual speakers are given ~3-5 minutes to share their personal story.
• I wanted to see if you might be interested in participating and learning more. If so, I can provide additional details about these opportunities.

INTERCEPT RESUBMITS NEW DRUG APPLICATION TO FDA FOR OBETICHOLIC ACID IN PATIENTS WITH LIVER FIBROSIS DUE TO NASH

A bit of history may be helpful to many. Obeticholic Acid (OCA) was filed with FDA in 2019 but FDA didn't go through with a planned public meeting called an adcom and instead issued what they called a complete response letter in June of 2020 and raised questions about safety.  They did not reject the application they just punted.  The department was going through some reorganization at the time and from a patient perspective we did not believe the sequence was proper but we were stuck with it.  Most companies would have abandoned the effort at that point.  To their great credit Intercept believed in the drug and continued their studies to answer the safety questions that had been raised.  Now they have much more data and good evidence of safety which we believe should resolve that issue.

An important point that many don't realize is that OCA is already being used to treat liver disease. It is approved for PBC (primary biliary cholengitis) which is a disease of the bile distribution network of the liver which leads to fibrosis. Because of the success with that specific condition it was studied for the larger problem of NASH.  It is important to know that OCA was the first drug to meet an endpoint that FDA had set for NASH drugs. It was this combination of facts that led us as patients to believe in 2020 that we would have the first therapy for NASH.