NASH Challenges: Next Silent Epidemic
Advancement in novel therapy is very crucial to halt progression of chronic disease like Non-alcoholic steatohepatitis (NASH). A decade research didn’t materialize any progress when it comes to approval of novel therapies. Considerable amount of research effort is going into discovering genetically validated drug targets. Therapy continuously struggling to get appropriate treatment options.
Recently approved Rezdiffra (resmetirom), hopes to address high medical unmet need, has been approved for the treatment of adults with noncirrhotic non-alcoholic steatohepatitis (NASH) with moderate to advanced liver scarring (fibrosis). Such market development would provide some relief to NASH untreated patients.
Challenges to be address:
- Lack of Patient Enrolment During Clinical Trial: NASH is a advanced form of NAFLD (Non-alcoholic fatty liver disease), which is largely asymptomatic, hence doesn’t goes for diagnosis. Due to low diagnosis rate, its difficult to find out NASH patients for clinical studies.
- Complex Disease Pathophysiology: Several genetically validated targets have been identified in laboratories, validating them in clinical area facing huge challenges. Recent studies revealed, modulation of retinoic acid receptor-related orphan receptor alpha (RORa) is a novel target
- Under diagnosis: Large pool of patients goes undiagnosed, largely attributed to lack of diagnosis tool, awareness, streamlined procedure of diagnosis, and misinterpretation of disease symptoms.
- Stringent Regulatory Process for Approval: Requirement of regulatory bodies, varies depending on country, for instance, EMA required both end points should meet. FDA required, improvement in fibrosis on various scale along with primary endpoints. NASH therapy landscape is sharing huge interest in failure to secure regulatory approval. Last year, in June, 2023, FDA rejected Intercept's Ocaliva NDA.
Way Forward: Having in-depth analysis on novel targets, and drug reaching in late clinical phase, need to be watched out to look at the development in the field. Research have to cross the old decade boundary of generic drug targets, and start a fresh approach toward, development of genetically validated drug targets.
