Phase III Trials Reveal That IPF Patients Treated Continually with ESBRIET for Long-term Have Reduced Risk of Death

Results from phase III trials with Esbriet® (pirfenidone), a drug developed by Roche, were recently presented at the European Respiratory Society (ERS) congress held in Amsterdam from September 26-30, 2015. The results revealed that Esbriet® is beneficial for long-term treatment of idiopathic pulmonary fibrosis (IPF).

Idiopathic pulmonary fibrosis is characterized by progressive scarring of lung tissue, which results in degradation of lung function. The disease often occurs in adults between 50 to 70 years old with a particular history of cigarette smoking. Patients with IPF may suffer from a variety of symptoms including shortness of breath, coughing, a crackling sound in the lungs during inhalation, and clubbing of the digits. More importantly, IPF often yields chronic oxygen deficiency in blood that may impact the function of many vital organs like the heart and the brain. The exact causes of IPF are still unknown, but environmental factors like cigarette smoking and exposure to various substances such as silica, metal dust, wood dust, coal dust, stone dust, biologic dusts, agricultural/farming products, and viral infections have been suggested as increasing risk of developing IPF. IPF affects approximately 100,000 individuals in the US and 110,000 in Europe. Men are more affected than women and the estimated median survival time following diagnosis is about 2 to 5 years.

From the therapeutic viewpoint, a number of medications are currently available on the market for treatment of IPF. The main purpose of these drugs is primarily to reduce symptoms, stop the disease from progression, preventing acute exacerbations, and extending survival time. These drugs have variable efficiencies, side effects and survival rates. Esbriet is an oral drug available in more than 38 countries worldwide, and the company is planning to expand access to the drug in more than 55 countries. It is approved for treatment of IPF in many countries in Europe four years ago, and the US a year ago. Esbriet has been shown to have a well-established safety profile. As a result, more than 20,000 IPF patients worldwide have been treated with Esbriet for the equivalent of a year. However, long term consequences of the drug on patients with IPF were unknown and in Phase III trials, Roche extended treatment of IPF patients with Esbriet for 2 years.

In phase III trials (ASCEND and CAPACITY I and II), a total of 1,247 patients participated. The results suggested that after one year of treatment, Esbriet is shown to slow down loss of lung function by about 48%. After two years (120 weeks) of treatment with Esbriet, the results revealed that risk of death in IPF patients reduced by 38% when compared to placebo. The latter constitutes the first long-term results showing continued attenuation in risk of death for patients with IPF. Analysis of data suggested that Esbriet is also beneficial for treatment after hospitalization. When compared to placebo, it was observed that patients hospitalized within the first six months of treatment had decreased disease progression by more than 10% and risk of death reduced by more than two-thirds when treated with Esbriet for one year. The latter demonstrated that continuing treatment with Esbriet after early hospitalization could slow down disease progression.

In conclusion, Esbriet® continues to demonstrate safety and efficacy in treating IPF. The drug is shown to slow down loss of lung function and reduce risk of death of IPF patients treated continually for the long term. Because only limited information is currently available for clinical decisions related to treatment, these findings are of particular value for physicians and patients.

“These new data demonstrate Esbriet’s ability to reduce the risk of death for patients with this severe, progressive lung disease,” said Sandra Horning, M.D., Roche’s Chief Medical Officer and Head of Global Product Development. “As the first long-term mortality data in IPF patients treated with Esbriet, these results provide valuable information to help physicians and patients make decisions about their treatment.”