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FDA Approves Esbriet to Treat Idiopathic Pulmonary Fibrosis

Progressive scarring of lungs leaves patients short of breath

Pirfenidone (Esbriet, InterMune, Inc.) has received FDA approval for the treatment of idiopathic pulmonary fibrosis (IPF), a condition in which the lungs become progressively scarred over time.

Patients with IPF experience shortness of breath, cough, and have difficulty participating in everyday physical activities. Other treatments for IPF include oxygen therapy, pulmonary rehabilitation, and lung transplant.

The FDA granted pirfenidone fast track, priority review, orphan product, and breakthrough designations and approved it ahead of the prescription drug user fee goal of November 23, 2014.

Pirfenidone’s safety and effectiveness were established in three clinical trials of 1,247 patients with IPF. The decline in forced vital capacity (FVC) — the amount of air that can be forcibly exhaled from the lungs after taking the deepest breath possible — was significantly reduced in patients receiving pirfenidone compared with patients receiving placebo.

The ASCEND trial, a placebo-controlled, randomized, double-blind, phase III study, 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 at 52 weeks, when 17% of patients who received pirfenidone had a decline in lung function (measured by a decline in FVC of at least 10%) compared with 32% of patients who received placebo.

Pirfenidone’s mechanism of action is not understood, although it is believed to interfere with the production of transforming growth factor-beta, a small protein in the body involved in how cells grow, and tumor necrosis factor-alpha, a small protein involved in inflammation.

Pirfenidone is not recommended for patients who have severe liver problems or end-stage kidney disease or who require dialysis. It should be taken with food to minimize the potential for nausea and dizziness. Patients should avoid or minimize exposure to sunlight and sunlamps and wear sunscreen and protective clothing, as pirfenidone may cause patients to sunburn more easily.

The most common side effects of Esbriet are nausea, rash, abdominal pain, upper respiratory tract infection, diarrhea, fatigue, headache, dyspepsia, dizziness, vomiting, decreased/loss of appetite, gastro-esophageal reflux disease, sinusitis, insomnia, decreased weight, and arthralgia.

Sources: FDA; October 15, 2014; and Intermune; October 15, 2014

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