Pfizer has received approval from the US Food and Drug Administration (FDA) for its Rapamune (sirolimus) to treat patients with lymphangioleiomyomatosis (LAM).
LAM is a rare and progressive disease, which affects the lungs, kidneys and the lymphatic system.
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Pfizer collaborated with the FDA, University of Cincinnati College of Medicine critical care and sleep medicine pulmonary director Dr Francis X. McCormack, and the LAM Foundation to assess Rapamune as a treatment option for LAM in the US.
Pfizer global innovative pharmaceuticals business global medical affairs head and senior vice-president Dr Rory O’Connor said: "Pfizer is proud to gain approval for Rapamune as the first treatment for patients with LAM, through our work with the FDA, the clinical investigation team and the LAM Foundation.
"This type of cooperative effort creates opportunities for innovation in developing therapies for patients with rare diseases."
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The approval was based on results of multi-centre international lymphangioleiomyomatosis efficacy of sirolimus (MILES) trial, which comprised 89 LAM patients with moderate lung impairment.
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By GlobalDataAccording to the firm, the trial demonstrated that those treated with Rapamune for one year experienced stabilisation of lung function measured by forced expiratory volume in one second (FEV1).
In the US, Rapamune also received approval as an immunosuppressive agent for the prophylaxis of organ rejection in kidney transplant patients aged 13 years and older.
It is prescribed to kidney transplant patients to prevent their natural immune system from rejecting the transplanted kidney.
Image: Low-magnification micrograph of lymphangioleiomyomatosis. Photo: courtesy of Nephron.
