Pfizer gets first approval for rare lung disease
pharmafile | May 29, 2015 | News story | Sales and Marketing | FDA, Pfizer, Rapamune, lung disease
Pfizer’s Rapamune will become the first drug available to treat the very rare lung disease lymphangioleiomyomatosis (LAM) after receiving approval from the FDA.
LAM is thought to affect only between two and five women per million worldwide of childbearing age, and is characterised by an abnormal growth of smooth muscle cells that invade lung tissues, blocking the airways, blood vessels and lymph vessels.
This obstructs airflow and limits the supply of oxygen to the body. The symptoms are similar to other lung conditions such as asthma, emphysema and bronchitis.
Rapamune (sirolimus) received priority review and orphan drug designation for the disease in February. At the time, Susan Sherman, executive director of the LAM Foundation, said: “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.”
The FDA’s approval is based on results from a trial of 89 LAM patients that showed that Rapamune slowed the decline in lung function over 12 months compared to a placebo. The most commonly reported side effects included chest pain, upper respiratory tract infection, dizziness, muscle pain and elevated cholesterol. Serious side effects including hypersensitivity and swelling have been observed in patients using the drug for other indications.
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.
Rapamune was originally approved in 1999 as an immunosuppressive agent to help prevent organ rejection in kidney transplant patients, for which it also received a priority review as well as breakthrough therapy designation. It is produced by Pfizer’s subsidiary Wyeth Pharmaceuticals, which the US firm bought in 2009 for $68 billion.
George Underwood
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