Rapamune (sirolimus) is FDA approved for lymphangioleiomyomatosis- Pfizer

Pfizer Inc.announced that the FDA has approved Rapamune (sirolimus) for the treatment of lymphangioleiomyomatosis (LAM), a rare, progressive disease that affects the lungs, kidneys and the lymphatic system. This is the first approved treatment that helps stabilize lung function in patients with LAM.

LAM is a rare progressive lung disease that usually affects women during their childbearing years and can result in abnormal growth of smooth muscle cells in the lung. Over time, the muscle growth can cause airway obstructions and limit the delivery of oxygen to the body. Approximately 800 patients in the U.S. are currently diagnosed with LAM, which is a rare disease and is often fatal. Rapamune is also approved in the U.S. as an immunosuppressive agent for the prophylaxis of organ rejection in kidney transplant patients aged 13 years and older. The FDA approval is based on the results from the Multicenter International Lymphangioleiomyomatosis Efficacy of Sirolimus or MILES Trial. The MILES Trial included 89 LAM patients with moderate lung impairment and showed that those treated with Rapamune for one year experienced stabilization of lung function measured by forced expiratory volume in one second (FEV1). The adverse drug reactions observed in this trial were consistent with the known safety profile for renal transplant patients receiving Rapamune, with the addition of weight decreased, which was reported at a greater incidence with Rapamune versus placebo. .