Genentech’s Esbriet Granted Breakthrough Therapy Designation for Unclassifiable Interstitial Lung Disease

Genentech's Esbriet Granted Breakthrough Therapy Designation for Unclassifiable Interstitial Lung Disease

Genentech announced that the U.S. FDA has granted Breakthrough Therapy Designation to Esbriet (pirfenidone) for adults with unclassifiable interstitial lung disease (uILD). The designation is based on results from a Phase II trial, which suggested Esbriet slowed disease progression in patients with uILD at 24 weeks.

Breakthrough Therapy Designation is designed to accelerate the development and review of medicines intended to treat serious or life-threatening conditions with preliminary evidence that indicates they may demonstrate a substantial improvement over existing therapies.

The Phase II data supporting Breakthrough Therapy Designation was recently presented as a late-breaking abstract at the 2019 European Respiratory Society’s annual meeting and simultaneously published in The Lancet Respiratory Medicine.

This international, multicenter, double-blind, randomized, placebo-controlled trial at 70 centers included patients (aged ≥18-85 years) with progressive fibrosing uILD, a percent predicted forced vital capacity (FVC) of 45% or higher and percent predicted carbon monoxide diffusing capacity (DLco) of 30% or higher, more than 10% fibrosis on high-resolution CT, and a high-resolution CT from the previous 12 months. The primary endpoint was mean predicted change in FVC from baseline over 24 weeks, measured by daily home spirometry. Analysis of the primary endpoint was affected by intraindividual variability in home spirometry values, which prevented application of the prespecified statistical model to the primary endpoint assessment. Over 24 weeks, predicted median change in FVC measured by home spirometry was -87.7 mL in the pirfenidone group versus -157.1 mL in the placebo group. The most common treatment-related treatment-emergent adverse events were gastrointestinal, fatigue, and rash.

About Esbriet (pirfenidone)

Esbriet (pirfenidone) is an orally available pyridone derivative that has anti-fibrotic and anti-inflammatory effects. It was approved for use in Europe in 2011 in adults with mild-to-moderate IPF, and in the U.S. in people with IPF in October 2014.

About Interstitial lung disease (ILD)

ILD is a term that broadly describes a diverse group of more than 200 types of rare pulmonary diseases. While ILDs share similar features, including cough and shortness of breath, each ILD has different causes, treatment approaches, and outlooks. Approximately 10% of people living with ILD reviewed by a multidisciplinary team cannot be given a definitive diagnosis, even after a thorough investigation, and in these cases, people are categorized as having unclassifiable interstitial lung disease (uILD).