WASHINGTON The Food and Drug Administration has approved an experimental drug from Raptor Pharmaceutical Corp to treat a rare genetic disorder that can cause irreversible tissue damage, organ failure and premature death.
Raptor's shares rose as much as 15.6 percent on Tuesday.
The drug, Procysbi, is designed to treat nephropathic cystinosis, the most common form of a disease known as cystinosis, in which toxic levels of cystine, a naturally occurring amino acid, build up in the body's cells and organs. The disease, which affects some 500 patients in the United States, is usually fatal by the age of 10.
Procysbi is a delayed-release formulation of cysteamine bitartrate, a drug sold under the brand name Cystagon by Mylan Inc. The drugs work by lowering cystine levels, potentially delaying kidney and other damage. Mylan's product is the current standard of care.
Raptor's drug is designed to be given once every 12 hours, instead of once every six hours for Cystagon.
The company's shares were up 7.3 percent to $7.03 in afternoon trading on Nasdaq. Earlier they rose as high as $7.67.