Axcella Therapeutics, a clinical-stage biotechnology company pioneering a new approach to treat complex diseases using multi-targeted endogenous metabolic modulator (EMM) compositions, announced that the U.S. Food and Drug Administration (FDA) has granted a Fast Track Designation to AXA1125 for the treatment of non-alcoholic steatohepatitis (NASH) with liver fibrosis. AXA1125 is a multi-targeted oral drug candidate that is currently being investigated in the EMMPACT Phase 2b clinical trial in NASH (NCT04880187) and a separate Phase 2a clinical trial in Long COVID (NCT05152849).
“Despite the fact that NASH is an epidemic that is impacting tens of millions of people here in the U.S. alone, there are no approved treatments today,” said Axcella President and Chief Executive Officer Bill Hinshaw. “We are pleased that, after reviewing the compelling data from our NASH program including those from two prior clinical studies, the FDA recognized AXA1125’s potential to address the needs of patients with this serious and chronic disease. We plan to leverage Fast Track to expedite our development path as we seek to provide an important first-line treatment option.”
Fast Track is a process designed by the FDA to facilitate the development and expedite the review of drugs to treat serious or life-threatening conditions with unmet medical needs. Drugs receiving Fast Track Designation may benefit from:
- More frequent meetings and written communications with FDA to discuss development plans,
- Accelerated Approval and Priority Review, assuming relevant criteria are met, and
- A Rolling Review of the drug’s New Drug Application (NDA) submission.
NASH is the most severe form of fatty liver disease that is driven by multifactorial systemic dysregulation of pathways associated with metabolism, inflammation and fibrosis. If left untreated, NASH may ultimately lead to life-threatening conditions such as cirrhosis or liver cancer. According to the Global Liver Institute’s U.S. NASH Action Plan, up to 40 million people in the U.S. alone are living with NASH and approximately 10% of U.S. children are afflicted with this disease. Incidence is expected to continue increasing in parallel with the obesity and type 2 diabetes epidemics.