Biodexa Receives Orphan Drug Designation From European Commission For eRapa In The Treatment Of Familial Adenomatous Polyposis (FAP)

Biodexa Pharmaceuticals PLC, a clinical-stage biopharmaceutical company focused on developing innovative treatments for diseases with unmet medical needs, has announced that the European Commission (EC) has granted Orphan Drug Designation for eRapa in the treatment of familial adenomatous polyposis (FAP). FAP is a largely inherited, precancerous disease of the colon, for which there is currently no approved pharmaceutical treatment.

Orphan Drug Designation is awarded by the European Commission following a positive opinion from the European Medicines Agency’s (EMA) Committee for Orphan Medicinal Products (COMP). This designation aims to encourage the development of medicines that can provide significant benefits to patients suffering from rare, life-threatening diseases. If eRapa is approved for marketing, the designation will grant it 10 years of marketing exclusivity in the EU. It will also provide additional incentives for sponsors, including eligibility for protocol assistance and potential exemptions or reductions in certain regulatory fees.

Stephen Stamp, CEO and CFO of Biodexa Pharmaceuticals PLC, said in a statement, “This Orphan Drug Designation is another important step as we move our FAP program forward into a registrational Phase 3 study. We are committed to providing global access to eRapa for as many patients as may benefit. This designation is an important step in our collaboration with EMA for our marketing authorization in the EU.”

Biodexa is currently finalizing the Phase 3 study of eRapa in FAP, which will be a double-blind, placebo-controlled trial involving 168 patients, with a 2:1 randomization of drug to placebo. The study is expected to take place across approximately 30 clinical sites in the US as well as Europe. The US component of the trial will be conducted by LumaBridge, based in San Antonio, Texas, while the European component will be managed by Precision for Medicine LLC. The Phase 3 study is supported by a $17.0 million grant from the Cancer Prevention Research Institute of Texas (CPRIT), with an additional $8.5 million matching contribution from the company, which has already been fully paid into escrow.