Experimental Treatments

Diflunisal is a nonsteroidal anti-inflammatory medication approved for mild to moderate pain and some forms of arthritis that’s been investigated as a potential FAP treatment. The oral therapy was shown to stabilize the transthyretin (TTR) protein, which is expected to prevent the formation of the toxic TTR clumps that drive FAP progression. It is sometimes used off-label in FAP as it was shown to slow disease progression in this patient population within a Phase 2/3 clinical trial.

CRX1008 (tolcapone) is an oral therapy that’s being developed to slow or prevent FAP progression. Tolcapone, its active ingredient, is already approved as an add-on therapy for Parkinson’s disease, and was shown to stabilize the TTR protein in a proof-of-concept Phase 2a trial of FAP patients.

Tafamidis meglumine is under investigation in the U.S. as an oral treatment for slowing FAP progression. It works by stabilizing the TTR protein and preventing the buildup of its toxic aggregates. While it is approved, under the brand name Vyndaqel, for adults with FAP in Europe, in the U.S. it is available only for adults with transthyretin amyloid cardiomyopathy, a FAP-related disease. It was shown to slow disease progression in multiple FAP Phase 2 and 3 trials.