Rapamune Approved for Rare Lung Disease

LAM affects mostly women of child-bearing age

By Scott Roberts

Friday, May 29, 2015

FRIDAY, May 29, 2015 (HealthDay News) -- Rapamune (sirolimus) has been approved by the U.S. Food and Drug Administration to treat a rare, progressive lung disease that mostly affects women of childbearing age.

Lymphangioleiomyomatosis (LAM) is characterized by the unusual growth of smooth-muscle cells among lung tissue. This can block normal airflow in the lungs and hinder delivery of oxygen to the rest of the body. The very rare disease affects only two to five women per million women globally, the FDA said in a news release.

The drug was first approved in 1999 to help prevent organ rejection among people aged 13 and older who had kidney transplant. It's now the first drug approved to treat LAM.

The FDA said it granted the drug's maker, Philadelphia-based Pfizer, financial incentives as part of a program to encourage development of medications to treat rare conditions.

The most common side effects reported during clinical testing included mouth and lip ulcers, diarrhea, abdominal pain, nausea, sore throat, acne and chest pain. More serious adverse effects included allergic-like reactions and swelling.

HealthDay