FDA approves Pfizer drug as rare lung disease treatment

SILVER SPRING, Md. — The Food and Drug Administration Friday announced that it had approved Rapamune (sirolimus), distributed by Pfizer’s Wyeth Pharmaceuticals, as a treatment for the rare lung disease lymphangioleiomyomatosis (LAM). 

 

Rapamune — which can be a table or oral solution —  has been distributed since 1999, used primarily as an anti-rejection drug in kidney transplant patients. Recently, though, it was granted breakthrough therapy and orphan product designation by the FDA — the latter is given to drugs that treat rare diseases. Rapamune was given priority review for its use treating LAM and clinical trials were funded in part by money from the FDA’s Orphan Products Grant Program. 

 

“Different FDA programs, such as orphan product designation and breakthrough therapy designation, provide sponsors with financial incentives and access to increased interactions and advice from FDA to facilitate development and timely approval of innovative treatments for rare diseases, which might not otherwise be developed,” director of the FDA’s Center for Drug Evaluation and Research’s Office of New Drugs director John Jenkins, said. “These kinds of special programs make it possible for FDA to help drug manufacturers get life-saving drugs to the people who need them much more quickly.”

 

LAM affects about 2 to 5 women per million in the world, whose lung tissues are invaded by an abnormal growth of smooth muscle cells that limits the oxygen delivered to the body.