FDA approves Esbriet (pirfenidone) for the treatment of idiopathic pulmonary fibrosis (IPF) in the United States

• Approximately 100,000 people in the United States have IPF, an irreversible and fatal lung disease¹
• Esbriet approved under FDA’s breakthrough designation program

Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced that the U.S. Food and Drug Administration (FDA) has approved Esbriet (pirfenidone) as a treatment for idiopathic pulmonary fibrosis (IPF) in the United States. IPF is a fatal disease caused by progressive scarring (fibrosis) of the lungs, which makes breathing difficult and prevents the heart, muscles and vital organs from receiving enough oxygen to work properly. The disease can advance quickly or slowly, but eventually the lungs will harden and stop working altogether.²

“This is a historic day for the people and their families in the United States who live with this deadly, incurable disease,” said Sandra Horning, M.D., Chief Medical Officer and Head of Global Product Development. “With today’s approval of Esbriet in the United States, people with IPF finally have an FDA-approved medicine that may slow the worsening of the disease.”

The approval of Esbriet is based on data from a large, placebo-controlled Phase III study known as ASCEND and is supported by two other large Phase III trials known as CAPACITY 1 and 2. In the ASCEND study, more patients who received Esbriet had a delay in the decline of lung function compared to those who received placebo as defined by the primary endpoint of percent change in Forced Vital Capacity (FVC), a measure of how well the lungs work based on the amount of air one can exhale with force after inhaling as deeply as possible.

The most serious adverse events observed in people who received Esbriet compared to those who received placebo were: elevations in liver enzymes found in the blood (a sign of liver damage; 3.7 percent vs. 0.8 percent), sensitivity to light or rash (9.0 percent vs. 1.0 percent) and gastrointestinal (GI) side effects that caused 2.2 percent of patients to discontinue treatment compared to 1.0 percent of those who received placebo.

Esbriet approval and InterMune

The approval of Esbriet in the United States would not have been possible without the 12 years of effort by InterMune, a Brisbane, California-based biotechnology company that dedicated itself to developing medicines for IPF and other serious diseases related to fibrosis. On September 29, 2014, Roche announced that they completed the acquisition of InterMune after a definitive merger agreement on August 24, 2014.

“Until today, the 100,000 people with IPF living in the United States did not have an FDA-approved treatment,” said Jonathan Leff, M.D., Executive Vice President of Research and Development at InterMune. “Today’s approval would not have been possible without the courage of patients, their families and the medical community that participated in the clinical studies of Esbriet.”

References

1. United States National Library of Medicine website. “Idiopathic Pulmonary Fibrosis” http://ghr.nlm.nih.gov/condition/idiopathic-pulmonary-fibrosis. Accessed October 7, 2014
2. National Institutes for Health website. “Idiopathic Pulmonary Fibrosis” http://www.nhlbi.nih.gov/health/health-topics/topics/ipf/. Accessed October 2, 2014