For the first time, a drug is available to treat lymphangioleiomyomatosis (LAM), a rare progressive lung disease that primarily affects women of childbearing age.
The FDA in late May approved sirolimus, which is marketed as Rapamune, to treat LAM (http://1.usa.gov/1J5JLde). The drug originally was approved in 1999 as an immunosuppressive agent to help prevent organ rejection in patients 13 years or older who had undergone kidney transplantation. The agency gave sirolimus an expedited review because it can offer significant improvement for a serious disease. The medication also received orphan drug status because LAM is a rare illness. According to the US National Library of Medicine, only 2 to 5 women per 1 million women worldwide are known to have LAM.
Voelker R. Drug Approved for Rare Lung Disease. JAMA. 2015;314(1):17. doi:10.1001/jama.2015.7107