FDA Accepts SNDA for Priority Review of Rapamune (sirolimus) for Treatment of Lymphangioleiomyomatosis (LAM)

Pfizer has announced that the FDA has accepted for priority review a supplemental New Drug Application (sNDA) for Rapamune (sirolimus) for the treatment of lymphangioleiomyomatosis (LAM), a rare, progressive lung disease in women of childbearing age that is often fatal. With the Priority Review designation for the sNDA, Pfizer anticipates a decision in June of 2015 based on the anticipated Prescription Drug User Fee Act (PDUFA) action date.

“If approved, Rapamune would be the first FDA-approved treatment option for patients living with LAM,” said Steve Romano, SVP and head of Global Medicines Development at Pfizer’s Global Innovative Pharmaceuticals Business. “We look forward to continuing to work closely with the FDA throughout the review process.”

The sNDA is based on results from the Multicenter International Lymphangioleiomyomatosis Efficacy and Safety of Sirolimus (MILES) Trial. The MILES Trial included 89 LAM patients with moderate lung impairment who were randomized to receive Rapamune (dose adjusted to 5-15 ng/mL) or placebo for 12 months, followed by a 12 month observation period. In the trial, those treated with Rapamune for one year experienced stabilization of lung function as measured by forced expiratory volume in one second (FEV1). Full results of the MILES Trial were published in the New England Journal of Medicine. The most common adverse events reported during the study were mucositis, diarrhea, nausea, hypercholesterolemia, acneiform rash and swelling in the lower extremities. The adverse drug reactions observed were consistent with the known safety profile of Rapamune in renal transplant patients, with the exception of weight decreased, which was reported at a greater incidence with Rapamune compared to placebo.

“The results of the MILES Trial demonstrated that Rapamune has the potential to stabilize lung decline in patients suffering from LAM,” said Dr Francis X. McCormack, Director of Pulmonary, Critical Care and Sleep Medicine at the University Of Cincinnati School of Medicine and the lead investigator of the MILES Trial. “We are excited about the FDA’s review of Rapamune and the potential to make this medication available to LAM patients.”

The MILES trial was conducted by Dr McCormack and conducted within the NIH Rare Lung Diseases Consortium. Pfizer provided study drug and a portion of the funding, but had no involvement in the design or conduct of the study. The LAM Foundation assisted with the recruitment of patients and logistics for the study.

“For 20 years, the LAM Foundation has been committed to seeking treatment options for LAM and we are thrilled about the possibility of getting a therapy approved to treat this rare and potentially deadly disease,” said Susan E. Sherman, Executive Director of the LAM Foundation.