The Food and Drug Administration has approved Rapamune for the treatment of lymphangioleiomyomatosis (LAM), a rare, progressive lung disease, the agency announced May 28.
LAM primarily affects women of childbearing age and is characterized by abnormal growth of smooth muscle cells that can cause destruction of the lung, limiting the delivery of oxygen to the body.
Rapamune (sirolimus) was originally approved in 1999 as an immunosuppressive agent to help prevent organ rejection in patients 13 years and older who were receiving kidney transplants. The drug is available as both a tablet and an oral solution.
In a clinical trial, researchers measured the safety and efficacy of the drug for treatment of LAM by comparing Rapamune with placebo in 89 patients for a 12-month treatment period, followed by a 12-month observation period.
During the 12-month treatment period, the difference in the average decrease in FEV1 (the rate of change in how much air a person can exhale during a forced breath in 1 second) was approximately 153 mL. After discontinuation of Rapamune, the decline in lung function resumed at a rate similar to the placebo group.
Because there are no available alternatives approved for treatment of LAM, Rapamune received orphan product designation from the FDA, which provides grants for clinical studies on safety and/or effectiveness of drugs used to treat rare diseases such as LAM.
Common side effects associated with Rapamune for the treatment of LAM include mouth and lip ulcers, diarrhea, abdominal pain, nausea, sore throat, acne, chest pain, leg swelling, upper respiratory tract infection, headache, dizziness, muscle pain, and elevated cholesterol.
Rapamune is manufactured by Wyeth Pharmaceuticals, a subsidiary of Pfizer.
For more safety information, please visit the FDA website .
