FDA Grants Breakthrough Therapy Designation for Genentech’s Esbriet (pirfenidone) in Unclassifiable Interstitial Lung Disease

• There are currently no FDA-approved treatments for unclassifiable ILD (uILD), a debilitating, severe respiratory condition
• The designation is based on results from a Phase II trial, which suggested Esbriet slowed disease progression in patients with uILD at 24 weeks

SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)-- Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to Esbriet^® (pirfenidone) for adults with unclassifiable interstitial lung disease (uILD). The designation was granted based on data from a Phase II trial, which studied the efficacy and safety of Esbriet in uILD. The study represented the first randomized controlled trial to exclusively enroll patients with progressive fibrosing uILD.

“Today’s milestone for Esbriet builds on our continued commitment to improving the standard of care for people living with fibrotic lung diseases,” said Levi Garraway, M.D., Ph.D., chief medical officer and head of Global Product Development. “We look forward to discussing the data with the FDA with the hope of bringing our important medicine to those with uILD who are currently without a treatment option.”

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 uILD.

The Phase II data supporting Breakthrough Therapy Designation were recently presented as a late-breaking abstract at the 2019 European Respiratory Society’s annual meeting and simultaneously published in The Lancet Respiratory Medicine. The data suggested Esbriet slowed disease progression and supported its efficacy on a number of lung function parameters including forced vital capacity (FVC), in people with uILD. The safety and tolerability profile of Esbriet in people with uILD was comparable with that observed in Phase III trials in people with idiopathic pulmonary fibrosis (IPF).

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. This is the 33rd Breakthrough Therapy Designation for Genentech’s portfolio of medicines.

About the Phase II 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% 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. Secondary endpoints were change in FVC measured by site spirometry, proportion of patients who had a more than 5% or more than 10% absolute or relative decline in percent predicted FVC measured by clinic-based spirometry, change in percent predicted DLco, change in 6-min walk distance (6MWD), change in University of California San Diego-Shortness of Breath Questionnaire (UCSD-SOBQ) score, change in Leicester Cough Questionnaire score, change in cough visual analogue scale, and changes in total and subscores of the St. George’s Respiratory Questionnaire (SGRQ), all of which were compared with baseline.

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 (Q1-Q3 -338.1 to 148.6) in the pirfenidone 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 pirfenidone than placebo (treatment difference 95.3 mL, p=0.002). Results for DLco and 6MWD generally trended in favor of pirfenidone treatment. Adverse event reporting reflected the known safety profile of pirfenidone. The most common treatment-related treatment-emergent adverse events were gastrointestinal disorders (47% in the pirfenidone group vs 26% in the placebo group), fatigue (13% vs 10%), and rash (10% vs 7%). Pirfenidone treatment was associated with less loss to lung function and exercise capacity compared with placebo over 24 weeks. The results of this study suggest that patients with progressive fibrosing uILD may benefit from pirfenidone therapy.

About Esbriet

Esbriet is an oral medicine approved for the treatment of IPF and is available in more than 60 countries worldwide. Esbriet 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. In early 2017, the U.S. Food and Drug Administration (FDA) approved the Esbriet 801 mg and 267 mg tablets as new options for administering the medicine for the treatment of IPF. The 801 mg tablets, which are now available in the U.S., offer people with IPF a maintenance option for taking Esbriet with fewer pills per day.

What is Esbriet?

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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