The Development Landscape for Non-Alcoholic Steatohepatitis

The Development Landscape for Non-Alcoholic SteatohepatitisThe Development Landscape for Non-Alcoholic Steatohepatitis Q&A

Q&A with Claudia Filozof, MD, PhD, Senior Medical Director in the Phase II-IV Cardiovascular/Metabolic Group and Richard Williams, PhD, JD, Executive Strategist,  Global Regulatory Affairs

Is this a good time to develop a NASH drug, and if so, why?

Claudia: Yes, it’s a very good time. There are multiple companies starting NASH development, and there is a huge unmet medical need with no treatment approved so far. Health authorities also have shown a lot of interest in supporting companies to speed up development. If you have the right compound, there are multiple advantages to starting a NASH program.

Richard: The cost to society will be enormous if we can’t treat this disease. I think most pharmaceutical companies, big and small, see this as a substantial unmet medical need. With this unmet medical need,  regulatory agencies can grant  conditional approval (accelerated approval in the US), where the drug is approved for marketing with the condition that the company later shows it has a clinical benefit based on clinical  outcomes. Continue reading

Non-Alcoholic Steatohepatitis: Limited Available Treatment Options but Promising Drugs in Development and Recent Progress Towards a Regulatory Approval Pathway

Abstract

The prevalence of non-alcoholic fatty liver disease (NAFLD) and non-alcoholic steatohepatitis (NASH) is increasing world-wide in parallel to the increase of the obesity epidemic. Insulin resistance (IR) and the accumulation of triglyceride-derived 103072_liver bw1toxic lipid metabolites play a key role in its pathogenesis. Multiple biomarkers are being evaluated for the non-invasive diagnosis of NASH. However, a percutaneous liver biopsy is still the gold standard method; the minimal diagnostic criteria include the presence of >5% macrovesicular steatosis, inflammation, and liver cell ballooning. Several pharmaceutical agents have been evaluated for the treatment of NASH; however, no single therapy has been approved so far. Due to the increasing prevalence and the health burden, there is a high need to develop therapeutic strategies for patients with NASH targeting both those with early-stage disease as well as those with advanced liver fibrosis.
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