Pfizer announced that the Food and Drug Administration (FDA) has approved Rapamune (sirolimus), the first drug approved to stabilize lung function in patients with lymphangioleiomyomatosis (LAM).

The safety and efficacy of Rapamune was established in the Multicenter International Lymphangioleiomyomatosis Efficacy of Sirolimus (MILES) Trial of 89 LAM patients with moderate lung impairment over a 12-month treatment period, followed by a 12-month observation period. The average decrease in forced expiratory volume in one second (FEV1) was approximately 153mL in the Rapamune group and after discontinuation, the decline in lung function resumed at a rate similar to the placebo arm.

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Rapmune is an immunosuppressant that inhibits T-lymphocyte activation and proliferation that occurs in response to antigenicand cytokine (Interleukin [IL]-2, IL-4, and IL-15) stimulation by a mechanism that is distinct from that of other immunosuppressants; it also inhibits antibody production. It is already indicated for organ rejection prophylaxis in renal transplant patients in combination with cyclosporine and corticosteroids in patients aged ≥13 and is available as 0.5mg, 1mg, and 2mg tablets in 100-count bottles and 10 blister cards of 10 tablets each, as well as a 60mg/60mL oral solution.

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