Biodexa Receives US FDA Fast Track for eRapa in Familial Adenomatous Polyposis Treatment
Tuesday, February 11, 2025
Biodexa Pharmaceuticals has received Fast Track designation from the US Food and Drug Administration (FDA) for eRapa, an encapsulated form of rapamycin.
FAP is a hereditary condition causing excessive polyp growth in the colon or rectum, typically emerging in adolescence. Without treatment, it leads to colorectal cancer. There is no approved drug therapy for FAP, and patients rely on regular monitoring and surgery.
The condition affects an estimated one in 5,000 to 10,000 people in the US and one in 11,300 to 37,600 in Europe. Biodexa has already secured Orphan Drug designation for eRapa in the US and intends to pursue a similar status in Europe.
eRapa is an oral tablet formulation of rapamycin (sirolimus), an mTOR inhibitor that plays a key role in regulating cell growth and metabolism.
mTOR is overexpressed in FAP polyps, making it a promising target for treatment. Rapamycin is already approved for preventing organ rejection in kidney transplant patients, but existing formulations have limitations, including poor bioavailability and variable pharmacokinetics.
eRapa utilises nanotechnology and pH-sensitive polymers to enhance absorption and reduce toxicity.
The designation aims to accelerate the development and review process for drugs addressing serious conditions with limited treatment options.
The decision was based on the potential of eRapa to meet the medical needs of patients with familial adenomatous polyposis (FAP), a genetic condition that leads to colorectal cancer if left untreated.
Currently, surgical removal of the colon and/or rectum is the only available treatment.
Phase 2 trial data demonstrated that eRapa was safe and well tolerated, showing a median 17% reduction in total polyp burden after 12 months, with a 75% non-progression rate.
Patients receiving an optimised dosage regimen experienced an 89% non-progression rate and a 29% median reduction in polyp burden. This regimen will be used in the upcoming Phase 3 study.
The formulation is protected by patents extending until 2035, with additional applications potentially extending protection further.
Source: globenewswire.com
