FDA Approves Treatment for Rare Lung Disease

May 28, 2015
Meghan Ross, Associate Editor

The FDA today approved sirolimus, the first drug to treat lymphangioleiomyomatosis, a rare lung disease.

The FDA today approved sirolimus (Rapamune), the first drug to treat lymphangioleiomyomatosis (LAM), a rare lung disease.

The FDA’s nod was based on a trial that examined Rapamune compared with placebo in 89 patients over a 1-year period, followed by a 1-year observation period. Researchers studied the patients’ forced expiratory volume in 1 second after 1 year and found that the patients taking the active treatment had a slower decline in lung function compared with those on placebo.

“After discontinuation of Rapamune, the decline in lung function resumed at a rate similar to the placebo group,” according to the FDA.

The treatment was made by Wyeth Pharmaceuticals Inc, a subsidiary of Pfizer, Inc, and it is available in both tablet form and as an oral solution.

Patients may experience adverse side effects such as mouth and lip ulcers, diarrhea, abdominal pain, nausea, sore throat, acne, chest pain, and leg swelling.

Rapamune’s history dates back to 1999, when it was first approved to be used as an immunosuppressive agent for patients receiving kidney transplants.

Women of childbearing age are particularly affected by LAM. However, the disease is very rare. The US National Library of Medicine states that between 2 and 5 women per million women are known to have this disease.