Biodexa Receives Orphan Drug Designation in Europe for eRapa in FAP

Core Viewpoint - Biodexa Pharmaceuticals has received Orphan Drug Designation in Europe for eRapa in familial adenomatous polyposis (FAP), following a similar designation from the FDA in 2019, and is preparing to initiate a Phase 3 study targeting a market opportunity of approximately $7.3 billion [1][2][5]. Company Overview - Biodexa Pharmaceuticals PLC is a clinical stage biopharmaceutical company focused on developing innovative treatments for diseases with unmet medical needs, including eRapa for FAP and other products for type 1 diabetes and rare brain cancers [9][13]. Orphan Drug Designation - The Orphan Drug Designation in the EU is granted by the European Commission based on a positive opinion from the EMA Committee for Orphan Medicinal Products, aimed at encouraging the development of drugs for rare, life-threatening diseases [2]. Phase 3 Study Details - The Phase 3 study of eRapa in FAP will be a double-blind placebo-controlled trial involving 168 patients, with a 2:1 randomization of drug to placebo, conducted across approximately 30 clinical sites in the US and Europe [3]. Market Opportunity - The addressable market for eRapa in FAP is estimated at $7.3 billion, based on the prevalence of FAP and the adult populations in the US and Europe, with a median annual cost of approved non-biologic orphan drugs in the US being $206,176 [5]. FAP Disease Overview - Familial adenomatous polyposis (FAP) is characterized by the proliferation of polyps in the colon and rectum, typically diagnosed in mid-teens, with no approved therapeutic options currently available [4]. eRapa Product Information - eRapa is a proprietary oral formulation of rapamycin, an mTOR inhibitor, designed to improve bioavailability and reduce toxicity compared to existing forms of rapamycin [6][10]. Clinical Study Results - Data from the Phase 2 study of eRapa indicated a median 17% reduction in total polyp burden at 12 months and a 75% non-progression rate, with cohort 2 showing an 89% non-progression rate and a 29% median reduction in polyp burden [7].