FDA approves Pfizer's Rapamune for new use, to treat LAM

29 May 2015

The US Food and Drug Administration yesterday approved pharma giant Pfizer’s (NYSE: PFE) Rapamune (sirolimus) to treat lymphangioleimyomatosis, a rare, progressive lung disease that primarily affects women of childbearing age. This is the first drug approved to treat the disease, the agency noted.

LAM is characterized by an abnormal growth of smooth muscle cells that invade lung tissues, including the airways, and blood/lymph vessels that cause destruction of the lung, resulting in airflow obstruction, and limiting the delivery of oxygen to the body. LAM is a very rare disease. According to the US National Library of Medicine, only between two and five women per million women worldwide are known to have the disease.

Originally approved as immunosuppressive in 1999

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