Rapamune Receives Approval for Rare Lung Disease
May 28, 2015 – The U.S. Food and Drug Administration (FDA) approved Rapamune® (sirolimus – Pfizer) to treat lymphangioleiomyomatosis (LAM) following a priority review. LAM is a very rare disease of the lungs, almost exclusively in women, about two women to five women per million have it. In LAM, smooth muscle tissue that grows in the lungs clogs airways, blood vessels and lymph channels restricting breathing and eventually destroying lung function. Current treatment includes symptom relief with bronchodilators, fluid removal from the lungs and lung transplants.
Rapamune was initially approved over 15 years ago to help prevent rejection of transplanted kidneys, and is now the first treatment to slow the progression of LAM. In a two-year clinical study of 89 LAM patients, those receiving Rapamune had better lung function than patients in a placebo group. Breathing capacity for actively treated patients began to decrease following the discontinuation of Rapamune. Initial dosing for LAM is 2mg once daily by mouth. The dose should be adjusted to achieve minimum blood concentrations between 5 nanograms (ng) and 15ng.
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