- The FDA approves Pfizer's (NYSE:PFE) Rapamune (sirolimus) for the treatment of lymphangioleiomyomatosis (LAM), a rare, progressive lung disease almost exclusively affecting women caused by mutations in the TSC1 gene. The disorder is characterized by the overgrowth of abnormal smooth muscle-like cells in the lungs which leads to the formation of cysts and the destruction of normal lung tissue. It affects as many as 5M women worldwide.
- Rapamune was originally approved in 1999 as an immunosuppressive agent to help prevent organ rejection in kidney transplant patients. The FDA designated it an Orphan Drug for the treatment of LAM. As such, it has a seven-year period of market exclusivity for the indication.