Biodexa Gets EU Orphan Drug Status for eRapa in FAP

On May 12, 2025, Biodexa Pharmaceuticals PLC announced that the European Commission has granted Orphan Drug Designation for their drug eRapa, aimed at treating familial adenomatous polyposis (FAP), a genetic condition that predisposes individuals to colon cancer. This follows a similar designation by the U.S. Food and Drug Administration in 2019. The biopharmaceutical company is preparing to launch a Phase 3 registrational study to further evaluate eRapa's effectiveness in a market estimated to be worth $7.3 billion.

Stephen Stamp, CEO and CFO of Biodexa Pharmaceuticals, emphasized the significance of this European designation as the company advances its FAP program into late-stage clinical trials. The designation is a crucial part of Biodexa's strategy to collaborate with the European Medicines Agency (EMA) to secure marketing authorization in Europe. The Orphan Drug Designation is intended for drugs that provide treatment options for rare and life-threatening diseases. It offers benefits such as 10 years of market exclusivity upon approval, as well as potential regulatory fee reductions.

Biodexa’s upcoming Phase 3 study of eRapa will involve 168 patients in a double-blind, placebo-controlled trial conducted at around 30 clinical sites across the United States and Europe. The study design includes a 2:1 randomization for the drug versus placebo. The U.S. portion will be managed by LumaBridge, while Precision for Medicine LLC will oversee the European component. The trial is financially supported by a $17 million grant from the Cancer Prevention Research Institute of Texas and a matching $8.5 million investment from Biodexa.

Familial adenomatous polyposis is characterized by the development of numerous polyps in the colon and rectum, usually appearing during the teenage years. Without treatment, FAP often progresses to cancer. Currently, the standard management involves regular monitoring and surgical removal of polyps, as no approved drug treatments exist. The prevalence of FAP is estimated at one in 5,000 to 10,000 in the U.S. and one in 11,300 to 37,600 in Europe. The overexpression of mTOR in FAP-associated polyps has led to the exploration of mTOR inhibitors like eRapa as potential treatments.

eRapa, an oral tablet formulation of rapamycin, inhibits the mTOR pathway, which is implicated in cell growth and cancer formation. Rapamycin is already approved in the U.S. under the brand name Rapamune for use in kidney transplant patients. Biodexa’s eRapa formulation utilizes nanotechnology to enhance bioavailability, reduce variability in drug metabolism, and minimize toxicity. Earlier Phase 2 trials demonstrated that eRapa is safe and well-tolerated, with a 17% median reduction in polyp burden after 12 months. The upcoming Phase 3 trial will implement the dosing regimen found effective in previous studies.

Biodexa Pharmaceuticals, listed on NASDAQ, focuses on developing treatments for diseases with unmet medical needs. Its pipeline includes eRapa for FAP and other conditions, tolimidone for type 1 diabetes, and MTX110 for rare brain cancers. The company's proprietary drug delivery technologies aim to improve the bioavailability and distribution of drugs. With headquarters in Cardiff, UK, Biodexa is committed to advancing its innovative therapies to address significant health challenges.