- The FDA has accepted Pfizer's supplemental NDA for Rapamune. Rapamune's original indication is for prevention of organ prevention in kidney transplant patients aged 13 or older.
- Rapamune (sirolimus) is intended to treat lymphangioleiomyomatosis (LAM), a rare, progressive, often fatal lung disease that affects reproductive-aged women.
- The basis of the submission is the MILES trial, a placebo-controlled study, which includes 89 women with LAM treated with Rapamune.
In the MILES trial, Rapamune-treated women experienced stabilization of lung function based on forced expiratory volume in one second (FEV1). The study included a 12-month study period, followed by a 12-month observation period, during which time, the goal was to slow down the progression of LAM. Because there are very limited treatment options for women with LAM, Rapamune has been given a Priority Review designation, with a PDUFA date in June.