Pfizer Inc. recently announced that the FDA has approved RAPAMUNE® (sirolimus) as the first treatment option for stabilizing lung function in patients with lymphangioleiomyomatosis (LAM), a rare, progressive disease that affects the lymphatic system, lungs and kidneys.
“Pfizer is proud to gain approval for RAPAMUNE as the first treatment for patients with LAM, through our work with the FDA, the clinical investigation team and the LAM Foundation,” said Rory O’Connor, MD, senior vice president and head of Global Medical Affairs, Global Innovative Pharmaceuticals Business, Pfizer Inc. “This type of cooperative effort creates opportunities for innovation in developing therapies for patients with rare diseases.”
The condition commonly affects women during their childbearing years and can lead to an abnormal growth of smooth muscle cells in the lung. In the US, about 800 patients receive a LAM diagnosis yearly, and is often a fatal disease.
RAPAMUNE is a drug approved as an immunosuppressive for the prevention of organ rejection in patients over 13 years of age who need a kidney transplant.
The RAPAMUNE approval is the result of data from the Multicenter International Lymphangioleiomyomatosis Efficacy of Sirolimus, also known as the MILES Trial, which revealed that the drug is effective in stabilizing lung function in 89 patients with LAM who had moderate lung impairment. In terms of adverse events, the data from the trial showed that the drug has a similar efficacy as the one found in renal transplant patients receiving RAPAMUNE.
“I am thrilled for families living with LAM,” said Dr. Francis X. McCormack, director of Pulmonary, Critical Care and Sleep Medicine at the University of Cincinnati College of Medicine and lead investigator of the MILES Trial. “The courage of the women who enrolled in the MILES trial made this possible. I am proud of the 200 investigators, coordinators and nurses who participated in the MILES trial that enabled FDA approval, and I sincerely thank all who supported the study.”
Pfizer worked in collaboration with the FDA, Dr. McCormack and the LAM Foundation to evaluate RAPAMUNE as a therapy for patients with LAM. Dr. McCormack and the National Institutes of Health Rare Lung Diseases Consortium conducted the MILES trial.
“This approval is a landmark breakthrough for LAM patients to have access to this important treatment option,” said Susan E. Sherman, executive director of the LAM Foundation. “It is the result of decades of work by researchers and women of the LAM community who volunteered for this pivotal clinical trial.”