FDA approves pirfenidone (Esbriet) and nintedanib (Ofev) for IPF

The U.S. FDA approved the first two drugs proven to slow progression of idiopathic pulmonary fibrosis (IPF): pirfenidone (Esbriet by Roche, which bought Intermune) and nintedanib (Ofev by Boehringer Ingelheim) on Wednesday October 15. Both drugs will offer new hope for patients, and new pain to the insurance companies and the government who pay for them.

Pirfenidone and nintedanib were each shown to reduce the average decline in lung function in randomized clinical trials published earlier in 2014.

Both drugs are expected to be available in the U.S. within weeks.

Idiopathic pulmonary fibrosis, affects about 100,000 people in the U.S, with a high 5-year mortality among those with severe progressive disease. However, the severity and rapidity of progression of IPF vary widely, with a substantial proportion of patients never experiencing severe impairment or life-threatening declines in lung function.

“There’s a lot of enthusiasm and excitement within the patient community,” Daniel M. Rose, CEO of the Pulmonary Fibrosis Foundation told the New York Times, but he also expressed concern about both drugs' cost.

Esbriet / pirfenidone's expected cost will be just under $100,000 a year in the U.S. (more than double what it already sells for in Europe and Canada), according to Roche. Boehringer Engelheim has not announced nintedanib / Ofev's expected price tag.

Paul Fonteyne, president of Boehringer's U.S.-facing division, expressed confidence that payers would not balk at nintedanib's cost (whatever it will be), telling the NYT reporter “The medical need is such that I doubt there would be a prolonged discussion on this."

One outstanding controversy is whether Roche will be able to market pirfenidone/Esbriet as being a lifesaving drug, not just a lung-preserving one. Roche's metaanalysis of its three phase III trials on pirfenidone (CAPACITY 1 and 2 and ASCEND) showed a slight survival benefit from pirfenidone, but a separate analysis used by the FDA did not conclude there was statistical significance to any benefit.

Sources:

"FDA approves Esbriet to treat idiopathic pulmonary fibrosis," FDA website.

New York Times: "F.D.A. Approves First 2 Drugs for Treatment of a Fatal Lung Disease," Andrew Pollack.