Two recent studies demonstrate that pirfenidone (Esbriet, Genentech) prolongs life expectancy by 2.47 years for IPF patients and that Esbriet and nintedanib (Ofev, Boehringer Ingelheim) reduce the decline in lung function.
The FDA recently granted orphan drug approval to Uptravi (selexipag) tablets to treat adults with pulmonary arterial hypertension (PAH), a chronic, progressive, and debilitating rare lung disease that can lead to death or the need for transplantation.
Lymphangioleiomyomatosis (LAM) is a rare, progressive lung disease that primarily affects women of childbearing age. The FDA has approved sirolimus (Rapamune, Pfizer) as the first drug to treat LAM. Rapamune was originally approved to prevent organ rejection.