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Priority review for Pfizer’s lung drug

pharmafile | February 23, 2015 | News story | Sales and Marketing FDA, Pfizer, Rapamune, lung, lymphangioleiomyomatosis, sirolimus 

The US Food and Drug Administration FDA has accepted Pfizer’s lung drug Rapamune for priority review.

Rapamune (sirolimus) prevents organ rejection after a kidney transplant and if given the greenlight, will be used for the treatment of rare lung disease lymphangioleiomyomatosis (LAM) in women of childbearing age.

“If approved, Rapamune would be the first FDA approved treatment option for patients living with LAM,” says Steve Romano, who is the head of Pfizer’s medicines development group and speciality care unit.

The priority review is based on the results from an 89-patient strong trial which demonstrated that the tablet has the potential to stabilise lung decline in patients suffering from LAM.

The most common adverse effects reported during the study were mucositis, diarrhoea, nausea, hypercholesterolemia, acneiform rash and swelling in the lower extremities.

The drug has so far not proved safe and effective in people who have had liver or lung transplants, and it carries an increased risk of developing infections or certain cancers, especially lymphoma and skin cancers.

LAM is characterised by abnormal growth of smooth muscle-like cells which can cause airway obstruction and limit the delivery of oxygen to the body. According to the LAM Foundation an estimated 30,000 to 50,000 women worldwide have the rare lung disease.

Susan Sherman, who is the foundation’s executive director, concludes: “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.”

Tom Robinson

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