An accumulation of data from multiple different studies supports the use of pirfenidone (Esbriet, from Roche) to treat idiopathic pulmonary fibrosis. One study in Belgium and the Netherlands demonstrated strong safety and efficacy profiles, and two more studies in the United States suggested a reduction in disease progression following treatment.
Released this month, the article, “Early Experience of Pirfenidone in Daily Clinical Practice in Belgium and The Netherlands: A Retrospective Cohort Analysis” in the journal Advances in Therapy was a retrospective study of 63 patients with idiopathic pulmonary fibrosis. Patients had been treated with pirfenidone for at least one year and were evaluated for lung function every three months. Researchers conducting the study analyzed data from these follow-up appointments for significant changes in lung function.
A major improvement in disease progression was seen for this cohort of patients. Before treatment with pirfenidone, a subset of patients with available data had an average decline in percent predicted forced vital capacity (%FVC) of 4.8%, while after six months of treatment they had an average decline of only 0.8%. There was a similar benefit seen in terms of percent diffusing capacity of the lungs for carbon monoxide (%DLCO). These improvements were accompanied by only minimal side effects, with loss of appetite and nausea the most common side effects.
In a similar study, the Phase 3 clinical study ASCEND (Efficacy and Safety of Pirfenidone in Patients With Idiopathic Pulmonary Fibrosis), 555 patients were treated with either pirfenidone or placebo three times a day for a total of one year. The main metric of efficacy for this study was the same as the last: change in %FVC from baseline. The results, published in the article, “A Phase 3 Trial of Pirfenidone in Patients with Idiopathic Pulmonary Fibrosis,” appeared in The New England Journal of Medicine.
A higher proportion of patients treated with placebo saw a decrease in %FVC than did the patients treated with pirfenidone. Pirfenidone also seemed to reduce the risk of death due to idiopathic pulmonary fibrosis, as there was a significant difference in progression-free survival between the two groups.
“Pirfenidone, as compared with placebo, reduced disease progression, as reflected by lung function, exercise tolerance, and progression-free survival, in patients with idiopathic pulmonary fibrosis,” wrote the authors of the study. “Treatment was associated with an acceptable side-effect profile and fewer deaths.” With multiple studies supporting pirfenidone as a treatment for idiopathic pulmonary fibrosis, ongoing research continues to support pirfenidone as a viable therapy for IPF.