FDA Grants Priority Review to Genentech’s Esbriet (pirfenidone) for Unclassifiable Interstitial Lung Disease

Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), announced today that the U.S. Food and Drug Administration (FDA) has accepted the company’s supplemental New Drug Application (sNDA) and granted Priority Review for Esbriet^® (pirfenidone) for the treatment of unclassifiable interstitial lung disease (UILD). The FDA is expected to make a decision on approval by May 2021.

“Since its U.S. approval, Esbriet has become a standard of care for people living with idiopathic pulmonary fibrosis. However, significant unmet need remains in fibrotic lung diseases, including unclassifiable interstitial lung disease (UILD),” said Levi Garraway, M.D., Ph.D., 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.”

The sNDA is based on results from a pivotal, 24-week Phase II trial, which was the first randomized controlled study specifically designed and conducted solely in people with UILD. The data were presented as a late-breaking abstract at the 2019 European Respiratory Society’s annual meeting and simultaneously published in The Lancet Respiratory Medicine.

In 2020, the FDA granted Orphan Drug Designation and Breakthrough Therapy Designation to Esbriet for UILD.

About Unclassifiable Interstitial Lung Disease

Interstitial lung disease (ILD) broadly describes a diverse group of more than 200 types of rare pulmonary diseases. ILDs share similar features, including cough and shortness of breath. However, each ILD has different causes, treatment approaches, and outlooks. Approximately 1 in 10 people living with ILD cannot be given a definitive diagnosis, even after a thorough investigation, and in these cases, they are categorized as having unclassifiable interstitial lung disease (UILD).

About the Pivotal Study

This international, multicenter, double-blind, randomized, placebo-controlled Phase II trial at 70 centers included patients (aged ≥18-85 years) with progressive fibrosing UILD, a percent predicted forced vital capacity (FVC) of 45 percent or higher and percent predicted carbon monoxide diffusing capacity (DLco) of 30 percent or higher, more than 10 percent fibrosis on high-resolution CT, and a high-resolution CT from the previous 12 months.

The primary endpoint was mean change in FVC from baseline over 24 weeks, measured by daily home spirometry. Key secondary endpoints included change in FVC measured by site spirometry; proportion of patients who had a more than 5 percent or more than 10 percent absolute or relative decline in percent predicted FVC measured by clinic-based spirometry; change in percent predicted DLco; change in 6-minute walk distance (6MWD); and several patient reported outcomes; all of which were compared with baseline.

The planned statistical model to estimate mean FVC decline could not be applied due to a number of physiologically implausible outlier results and short observation time for a few patients. Over 24 weeks, predicted median change in FVC measured by home spirometry was -87.7 mL (Q1-Q3 -338.1 to 148.6) in the Esbriet group versus -157.1 mL (-370.9 to 70.1) in the placebo group. Over 24 weeks, predicted mean change in FVC measured by site spirometry was lower in patients given Esbriet than placebo (treatment difference 95.3 mL, p=0.002). Results for DLco and 6MWD generally trended in favor of Esbriet treatment. Adverse event reporting reflected the known safety profile of Esbriet in IPF. The most common treatment-related and treatment-emergent adverse events in the Phase II UILD study were gastrointestinal disorders (47% in the Esbriet group vs. 26% in the placebo group), photosensitivity (8% vs. 2%), rash (10% vs. 7%), dizziness (8% vs. 3%), weight decreased (8% vs. 1%) and fatigue (13% vs. 10%). Esbriet treatment was associated with less loss of lung function and exercise capacity compared with placebo over 24 weeks. The results of this study suggest that patients with UILD may benefit from Esbriet therapy.

About Esbriet

Esbriet is an oral medicine approved for the treatment of idiopathic pulmonary fibrosis (IPF) and is available in more than 60 countries worldwide. Esbriet was FDA-approved for use in people with IPF in October 2014.

Esbriet U.S. Indication

Esbriet is a prescription medicine used to treat people with a lung disease called idiopathic pulmonary fibrosis (IPF).

It is not known if Esbriet is safe and effective in children.

Select Important Safety Information

Before you take Esbriet, tell your doctor if you:

• have other medical conditions (particularly liver or kidney problems).
• are a smoker.
• are or plan to become pregnant or breastfeed (Esbriet has not been studied in these patients).
• are taking any prescription or over-the-counter medicines, vitamins, or herbal supplements.

What are the possible side effects of Esbriet?

Esbriet may cause serious side effects, including:

• liver problems. Patients should call their doctor if they have symptoms such as yellowing of the skin or eyes, dark or brown urine, pain on the upper right side of the stomach area, bleeding or bruising more easily than normal, or increased fatigue. A healthcare provider will also do regular blood tests to check the liver.
• sun sensitivity and rash. When patients are outside, they should use sunscreen (SPF 50) and wear a hat and clothes that cover the skin to avoid getting a sunburn.
• stomach problems. Esbriet may cause stomach problems such as nausea, vomiting, diarrhea, indigestion, heartburn, and stomach pain.

A healthcare provider may change the dose or discontinue Esbriet if side effects do not go away.

The most common side effects of Esbriet include upper respiratory tract infections, feeling tired, headache, dizziness, loss of appetite, sinusitis, insomnia, or weight loss.

These are not all the possible side effects of Esbriet.

What should patients avoid while taking Esbriet?

• Direct exposure to sunlight, or light from sunlamps and tanning beds.
• Other medicines that can make skin sensitive to sunlight.
• Smoking, which may affect how well Esbriet works.

Patients should call their doctor for medical advice about side effects. Patients may report side effects to the FDA at 1-800-FDA-1088 or http://www.fda.gov/medwatch or to Genentech at 1-888-835-2555.

Please see full Prescribing Information, including Patient Information, for additional important safety information at http://www.esbriet.com.

About Genentech

Founded more than 40 years ago, Genentech is a leading biotechnology company that discovers, develops, manufactures and commercializes medicines to treat patients with serious and life-threatening medical conditions. The company, a member of the Roche Group, has headquarters in South San Francisco, California. For additional information about the company, please visit http://www.gene.com.

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