The U.S. Food and Drug Administration offers various designations to drugs and drug developers to both incentivize medical advancements in the rare disease space and get treatments in the hands of people in need. One of these designations is known as Fast Track designation. This designation focuses on drugs that treat serious, rare, and life-threatening conditions and fill unmet needs (i.e. if there are no other available treatments or if other treatments show more efficacy). It also imbues the drug developer with benefits such as eligibility for Priority Review or Accelerated Approval. Healio recently shared that the FDA granted Fast Track designation to efinopegdutide, which is being developed for nonalcoholic steatohepatitis (NASH).
The Need for Better NASH Treatments
Nonalcoholic steatohepatitis is a liver disease that occurs in non-drinkers or people who drink very little. Fat accumulates in the liver, causing inflammation and damage. NASH can prevent the liver from working the way that it should. People with this condition may experience fatigue and weakness, weight loss, nausea and vomiting, itchy skin, and swelling of the legs and abdomen. Abdominal pain and jaundice (where your skin and eyes become yellow) may also occur.
Typically, NASH is treated with a variety of lifestyle changes. People are encouraged to abstain from drinking alcohol, lose weight, and exercise. Identifying and integrating more targeted treatments could change the landscape for people with NASH and allow them to live healthier, longer lives. Ideally, these therapies would also reduce fat accumulation and inflammation while improving liver function.
The FDA granted Fast Track designation to efinopegdutide, an investigational GLP-1 dual agonist, based on the findings from a Phase 2a clinical trial. 145 participants enrolled. The trial found that efinopegdutide showed improvement in liver fat levels. Moving forward, further studies are planned to identify how safe and effective efinopegdutide is in NASH control. 300 participants with pre-cirrhotic NASH (their livers have not become scarred yet) will enroll.