Pirfenidone Gets Priority Review for Unclassifiable Interstitial Lung Disease

The sNDA includes data from a phase 2 trial that evaluated the efficacy and safety of pirfenidone in patients with progressive fibrosing UILD.

The Food and Drug Administration (FDA) has granted Priority Review to pirfenidone (Esbriet; Genentech) for the treatment of unclassifiable interstitial lung disease (UILD).

The supplemental New Drug Application includes data from a double-blind, placebo-controlled phase 2 trial that evaluated the efficacy and safety of pirfenidone in patients with progressive fibrosing UILD (N=253). The primary efficacy end point was the mean predicted change in forced vital capacity (FVC) from baseline over 24 weeks, measured by daily home spirometry.

Findings from the trial showed the predicted median change in FVC measured by home spirometry was -87.7mL (Q1-Q3 -338.1 to 148.6) in the pirfenidone group vs -157.1mL (–370.9 to 70.1) in the placebo group; however, the planned statistical model to estimate mean FVC decline could not be applied due intraindividual variability and short observation time for some patients. An analysis of key secondary end points (ie, change in FVC measured by site spirometry, change in percent predicted carbon monoxide diffusing capacity, and change in 6-minute walk distance) did suggest a benefit with pirfenidone.

“Since its US approval, Esbriet has become a standard of care for people living with idiopathic pulmonary fibrosis,” said Levi Garraway, MD, PhD, chief medical officer and head of Global Product Development. “We are working closely with the FDA in hopes of offering Esbriet to people with UILD, a rare and debilitating disease.”

Pirfenidone was granted both Orphan Drug and Breakthrough Therapy designations for UILD. A decision on approval is expected by May 2021.

Esbriet is currently indicated for the treatment of idiopathic pulmonary fibrosis.


FDA grants Priority Review to Genentech’s Esbriet (pirfenidone) for unclassifiable interstitial lung disease. [press release]. San Francisco, CA: Genentech; January 21, 2021.