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FDA Approves Rapamune (Sirolimus) to Treat Rare Lung Disease

Immunosuppressive agent has been around since 1999

The FDA has given the green light to Rapamune (sirolimus, Wyeth Pharmaceuticals) for the treatment of lymphangioleiomyomatosis (LAM), a progressive lung disease that primarily affects women of childbearing age. It is the first drug approved to treat the disease.

LAM is characterized by an abnormal growth of smooth-muscle cells, which invade lung tissues as well as blood and lymph vessels, resulting in airflow obstruction and limiting the delivery of oxygen to the body. LAM is a very rare disease. According to the U.S. National Library of Medicine, only two to five women per million women worldwide are known to have the disease.

Rapamune (sirolimus), which is available as both a tablet and an oral solution, was originally approved in 1999 as an immunosuppressive agent to help prevent organ rejection in patients 13 years of age and older receiving kidney transplants.

Because the drug’s sponsor demonstrated that sirolimus may offer a substantial improvement over available therapies, it received a “breakthrough therapy” designation from the FDA. The drug also received “priority review” status, which allows the expedited review of drugs that have the potential to provide a significant improvement in safety or effectiveness in the treatment of a serious disease or condition. Moreover, sirolimus received an “orphan product” designation for its new indication because LAM is a rare disease or condition.

Sirolimus was compared with placebo in 89 patients with LAM for a 12-month treatment period, followed by a 12-month observation period. The study’s primary endpoint was the difference between the two groups in the rate of change in the forced expiratory volume in 1 second (FEV1). The difference in the average decrease in FEV1 during the 12-month treatment period was approximately 153 mL. After the discontinuation of sirolimus, the decline in lung function resumed at a rate similar to that of the placebo group.

The most common adverse events associated with sirolimus included 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 levels. Serious adverse events, including hypersensitivity and edema, have been observed in renal transplant patients.

Source: FDA; May 28, 2015.


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