Review of 'Drug repositioning framework driven by landscape analysis and competitive intelligence: A case report on Prader Willi Syndrome'

Drug repositioning is a promising strategy to develop therapeutics for rare diseases 1,2. In our present work, we explore a drug repositioning framework that can capture the clinical unmet needs of Prader Willi syndrome (PWS) showing ineffectiveness of GLP1 agonists 3, widely accepted as the standard of care for lifestyle obesity. Several therapeutic axes have been explored against PWS such as enzyme replacement therapy 4,5, Ghrelin analogues 6, MC4R agonist 7, however none of these approaches have shown preventive action, thus leaving a wide scope for exploring drug repositioning in PWS. Market share analysis of GLP1 agonists used against PWS along with the overall market size and growth of obesity medication shows the room for market penetrance in rare conditions of genetic obesity. Novo Nordisk and Eli Lilly dominate the obesity therapeutics market with their blockbuster drugs like Ozempic, Wegovy 8, Trulicity and most recently Zepbound 9. In addition, entry of Zealand Pharmaceuticals and Viking Therapeutics are set to provide additional therapeutic options to patients of obesity 10,11. However, the competitive landscape surrounding PWS therapies points to a significant gap, as genetic obesity has potential multi-systemic involvement and not only the metabolic axis 12. This framework provides life cycle management opportunities in other genetic obesity conditions such as Bardet-Biedl syndrome (BBS), Kleefstra syndrome- EHMT1 mutation, Alstrom syndrome, Biemond syndrome type 2, Wolfram syndrome, Cohen syndrome, familial isolated DCM and mitochondrial disorders 13.