The FDA has granted a new licence for Pfizer’s rare lung cancer drug Rapamune (sirolimus), the first time a treatment has gained approval for this form of the disease.
The US giant’s drug treats LAM a very rare, progressive lung disease that primarily affects women of childbearing age. This is the first drug approved to treat the disease.
LAM is characterised by an abnormal growth of smooth muscle cells that invade lung tissues, including the airways, and blood/lymph vessels that cause destruction of the lung, resulting in airflow obstruction, and limiting the delivery of oxygen to the body.
According to the US National Library of Medicine, only between two and five women per million women worldwide are known to have the disease.
Rapamune, which is available as both a tablet and an oral solution, was originally approved in 1999 as an immunosuppressive agent to help prevent organ rejection in patients 13 years and older receiving kidney transplants.
Pfizer‘s drug received both a breakthrough therapy designation and a priority review, which provides for an expedited review of drugs that have the potential to provide a significant improvement in treatment.
Rapamune also received orphan product designation for this indication because LAM is a rare disease or condition.
John Jenkins, director of the Office of New Drugs at the FDA, said: “Different FDA programmes, such as orphan product designation and breakthrough therapy designation, provide sponsors with financial incentives and access to increased interactions and advice from FDA to facilitate development and timely approval of innovative treatments for rare diseases, which might not otherwise be developed.
“These kinds of special programmes make it possible for FDA to help drug manufacturers get life-saving drugs to the people who need them much more quickly.”