Amicus Fights Its Way Through in Fabry's

Perseverance is a critical variable in drug discovery. Too little of it, and you are absolutely guaranteed to fail - no drug has ever made it to market without trying the patience of everyone involved. Too much of it, and you are very nearly guaranteed to waste all your money: most drug development projects don't work, and eventually reach a point where no amount of time or money could make them work, either. Many are the efforts where leaders have gritted their teeth, redoubled their efforts, and led everyone further into the abyss. But sometimes these things come through, and that's what seems to have happened with Amicus and their drug migalastat for Fabry's. It's a protein chaperone, one the the emerging class of drugs that work by stabilizing particular protein conformations to help regain function. At the end of 2012, Amicus and their partner GSK announced clinical trial results that didn't meet significance, which prompted GlaxoSmithKline to return rights to the drug to Amicus. Who kept on with it. And who announced today that the second Phase III study had come back positive, enough so that they plan to file for regulatory approval. (The belief is that the first Phase III enrolled an inappropriate mix of patients). Congratulations to the company, who may well have given many Fabry's patients their first opportunity for an oral therapy for their disease.
Source: In the Pipeline - Category: Chemists Tags: Clinical Trials Source Type: blogs