Biodexa Pharmaceuticals PLC, a clinical stage biopharmaceutical company developing a pipeline of innovative products for the treatment of diseases with unmet medical needs, announced the European Commission (EC) has granted Orphan Drug Designation for eRapa in familial adenomatous polyposis (FAP), a largely inherited precancerous disease of the colon for which there is currently no pharmaceutical intervention.
” This Orphan Drug Designation is another important step as we move our FAP program forward into a registrational Phase 3 study.” said Stephen Stamp, CEO and CFO of Biodexa Pharmaceuticals PLC. “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.”
Orphan Drug Designation in the EU is granted by the European Commission based on a positive opinion issued by the EMA Committee for Orphan Medicinal Products (COMP). It is intended to encourage the development of drugs that may provide significant benefit to patients suffering from rare, life-threatening diseases. If approved for marketing, this designation will provide 10 years of marketing exclusivity and also provide special incentives for sponsors, including eligibility for protocol assistance and possible exemptions or reductions in certain regulatory fees.
The Phase 3 study of eRapa in FAP is in the final stages of implementation. It will be a double-blind placebo-controlled trial in 168 patients, randomized 2:1 drug / placebo. It is expected the study will be conducted in approximately 30 clinical sites across the US and Europe. The US component of the study will be conducted by LumaBridge, based in San Antonio, Texas and the European component will be conducted 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”) and a Company match of $8.5 million which has already been paid, in full, into escrow.