On the verge of submitting a rare-disease drug for approval, Raptor Pharmaceuticals Corp. is counting on one major selling point: Its drug doesn’t stink.
The 14-person Novato company will file a new drug application early in first-quarter 2012 for delayed-release cysteamine, a treatment for a deadly inherited disease known as cystinosis. The drug uses the same active ingredient as Cystagon, approved in 1994 by the Food and Drug Administration.
The knock against Cystagon has been a putrid rotten egg smell and its every-six-hours dosing. That’s no small thing in a disease that can kill children by the age of 9 — parents must wake a child during the night to give the powder-filled capsule.