FDA Approves Esbriet® (pirfenidone) for the Treatment of Idiopathic Pulmonary Fibrosis (IPF) in the United States
Approximately 100,000 People in United States Have IPF, an Irreversible and Fatal Lung Disease1
Esbriet Approved Under FDA's Breakthrough Designation Program
Expected Availability in United States Within Two 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 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 (pronounced es-BREE-et) 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.
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."
Esbriet will be available to patients within two weeks. A comprehensive patient support program designed to help with access, financial support and ongoing education will be available for people who are prescribed Esbriet for IPF.
Approximately 100,000 people in the United States have IPF, an irreversible and ultimately fatal disease characterized by progressive loss of the ability of the lungs to absorb oxygen due to scarring.1,2 The cause is unknown and there is no cure. IPF inevitably causes shortness of breath and destruction of healthy lung tissue, although some people may experience periods of stability with the disease.2-5 The median survival time from diagnosis is two to five years, and the five-year survival rate is approximately 20 to 40 percent.2,6 IPF typically occurs in people over the age of 45, and tends to affect slightly more men than women.1
Esbriet is an oral medicine for the treatment of idiopathic pulmonary fibrosis. The mechanism of action of Esbriet is not understood, although it is believed to interfere with the production of Transforming Growth Factor (TGF)-beta, a small protein in the body involved in how cells grow and Tumor Necrosis Factor (TNF)-alpha, a small protein that is involved in inflammation. Earlier this year, the FDA granted Esbriet Breakthrough Therapy Designation based on the positive data from the ASCEND clinical trial and the serious and life-threatening nature of IPF. It has also been granted Orphan Drug designation in the United States.
Esbriet was developed for use by InterMune in the United States, Europe and other countries. It was granted marketing authorization in the European Union (EU) in 2011 for the treatment of adults with mild to moderate IPF in all 28 EU member countries, and has since been approved in Norway, Iceland and Canada.
Esbriet Efficacy in IPF
The efficacy and safety profile of Esbriet was studied in one of the largest Phase III programs to date in IPF and included three Phase III studies.
The ASCEND study was a multinational, multicenter, randomized, double-blind, Phase III study that enrolled 555 patients with mild to moderate IPF. The primary endpoint was the percent change in FVC from baseline to the end of the study, measured at 52 weeks.
The study showed that after one year 17 percent of patients who received Esbriet had a decline in lung function (measured by a decline in FVC of at least 10 percent) compared to 32 percent of patients who received placebo.
The CAPACITY studies were two concurrent, multinational, multicenter, randomized, double-blind, Phase III studies that enrolled collectively 779 patients with mild to moderate IPF. The primary endpoint was the percent change in FVC from baseline to the end of the study, at 72 weeks. Data from the CAPACITY trials and on survival for patients in the clinical trials of Esbriet are also included in the prescribing information.
Across the Phase III trials of Esbriet, the most common adverse events (observed in at least 10 percent of patients) are nausea, rash, abdominal pain, upper respiratory tract infection, diarrhea, fatigue, headache, dyspepsia (indigestion), dizziness, vomiting, anorexia, gastro-esophageal reflux disease, sinusitis, insomnia, weight decrease and arthralgia (joint pain).
Esbriet is a prescription medicine used to treat people with a lung disease called idiopathic pulmonary fibrosis (IPF).
Important Safety Information
Before taking Esbriet, patients should tell their doctor if they:
have liver problems
have kidney problems
are a smoker
have any other medical conditions
are pregnant or plan to become pregnant. It is not known if Esbriet will harm the unborn baby.
are breastfeeding or plan to breastfeed. It is not known if Esbrietpasses into breast milk. Patients and their doctor should decide if they will take Esbrietor breastfeed.
Patients should tell their doctor about all the medicines they take, including prescription and over-the-counter medicines, vitamins, and herbal supplements.
Patients should avoid:
Sunlight. Esbriet can make skin sensitive to the sun and the light from sunlamps and tanning beds. Patients could get a severe sunburn. Patients must use sunscreen (SPF50) and wear a hat and clothes that cover the skin if they have to be in sunlight. Patients should talk to their doctor if they get a sunburn.
Smoking. Smoking may affect how well Esbriet works.
Esbriet may cause serious side effects, including:
Liver problems: Patients must call their doctor right away if they have unexplained symptoms such as yellowing of their skin or the white part of their eyes (jaundice), dark or brown (tea colored) urine, pain on the upper right side of their stomach area (abdomen), bleeding or bruising more easily than normal, feeling tired. The doctor will do blood tests to check how a patient's liver is working during treatment with Esbriet.
Sensitivity to sunlight (photosensitivity) and rash.
Stomach problems. Esbriet may cause stomach problems such as nausea, vomiting, diarrhea, indigestion, heartburn, and stomach pain. Patients must tell their doctor right away if their stomach problems get worse or do not go away. The doctor may need to change the patient's dose of Esbriet.
The most common side effects of Esbriet include feeling tired, headache, dizziness and decreased or loss of appetite.
These are not all the possible side effects of Esbriet. For more information, patients should ask their doctor or pharmacist.
You may report side effects to the FDA at (800) FDA-1088 or http://www.fda.gov/medwatch. You may also report side effects to InterMune at (888) 486-6411.
Please visit http://www.Esbriet.com for the full Prescribing Information including Patient Information for additional important safety information.
Founded more than 35 years ago, Genentech is a leading biotechnology company that discovers, develops, manufactures and commercializes medicines to treat patients with serious or 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.
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.
3. Coalition for Pulmonary Fibrosis. Facts About Idiopathic Pulmonary Fibrosis. http://www.coalitionforpf.org/facts-about-idiopathic-pulmonary-fibrosis. Accessed August 14, 2014.
4. Kim DS, et al. Proc Am Thorac Soc. 2006;3:285-292.
5. Raghu G, et al. Am J Respir Crit Care Med. 2011;183:788-824
6. King TE Jr et al., Proc Am Thorac Soc. 2006;4:285-292.
Terence Hurley, 650-467-6800
Ekaterine Kortkhonjia, 650-467-5873
Karl Mahler, 011 41 61 687 8503