Roche’s ESBRIET (pirfenidone) Deemed Safe in Systemic Sclerosis Patients with Interstitial Lung Disease

Results showing the safety of Esbriet (pirfenidone) in treating interstitial lung disease due to systemic sclerosis were recently presented at the European League Against Rheumatism Annual European Congress of Rheumatology. The U.S. Food and Drug Administration approved Esbriet (pirfenidone), which is currently marketed by Roche, as a treatment for idiopathic pulmonary fibrosis (IPF) in the Fall of 2014. Now, researchers have evaluated the therapy’s safety in patients with interstitial lung disease, which can arise as a complication of systemic sclerosis.

Systemic sclerosis (SSc) is an autoimmune disease characterized by thickening of the skin due to accumulation of collagen (the main structural protein of the connective tissue) and can include damage to internal organs, including the lungs. A frequent complication of SSc is interstitial lung disease (ILD), which often progresses in SSc patients and has a poor prognosis. There are unmet therapeutic needs for treating ILD in systemic sclerosis, and researchers believe that Esbriet could offer promising therapeutic value.

The data is from an open-label, 16-week trial with patients with systemic sclerosis (SSc) and confirmed interstitial lung disease (ILD) (in total, 63 patients were diagnosed with ILD by high-resolution CT). Patient enrollment was based on the following eligibility criteria: a forced vital capacity (FVC) of at least 50%, diffusing capacity for carbon monoxide (DLCO) of 40% minimum, and disease for at least 7 years (FVC measures the change in lung volume between a full inspiration followed by forced air exhale; DLCO is one of the most clinically valuable tests of lung function and measures the ability of lungs to transfer gas from inhaled air to the red blood cells in pulmonary capillaries). Patients with pulmonary hypertension or severe gastroesophageal reflux disease were excluded from the study.

For the study, patients were randomly assigned to receive Esbriet at different dosages for 2 to 4 weeks. The primary outcome was to measure drug safety and was determined by laboratory tests, evaluating treatment-emergent adverse events (TEAEs) and monitoring vital signs with electrocardiogram (ECG).

The team found at week 16 that patients exhibited no alterations in vital signs or even in the laboratory test results. Only three patients registered serious adverse events — obstruction in the small intestine, bronchitis, pulmonary hypertension and worsening ILD. From the pool of patients who reported TEAEs (96.8%), 30.2% had mild TEAEs, 47.6% reported moderate TEAEs and only 19% reported severe TEAEs.

The team commented, “The observed [adverse events] were expected and consistent with those previously seen with pirfenidone treatment in [pulmonary fibrosis] trials. The data support further investigation of pirfenidone in SSc-ILD.”