Case Study: FDA approves Esbriet® (pirfenidone) to treat idiopathic pulmonary fibrosis

The U.S. Food and Drug Administration today approved Esbriet (pirfenidone) for the treatment of idiopathic pulmonary fibrosis (IPF).

Idiopathic pulmonary fibrosis is a condition in which the lungs become progressively scarred over time. As a result, patients with IPF experience shortness of breath, cough, and have difficulty participating in everyday physical activities. Current treatments for IPF include oxygen therapy, pulmonary rehabilitation, and lung transplant.

Developing new molecule to USFDA approval

InterMune licensed certain rights to pirfenidone from Marnac, Inc. and its co-licensor, KDL GmbH, in 2002. In 2007 the company purchased from Marnac and KDL the rights to sell the compound under the patents in the United States, Europe and other territories, except in Japan, Taiwan and South Korea, where rights to the molecule were licensed to Shionogi & Co. Ltd. of Japan. Pirfenidone is approved for marketing in 30 European countries (28 in EU plus Norway and Iceland) and Canada under the InterMune trade name Esbriet^® and in Japan and South Korea where it is marketed by Shionogi & Co. Ltd under the trade name Pirespa®. Under different trade names, pirfenidone is also approved for the treatment of IPF in China, India, and Argentina. Pirfenidone is not approved for marketing in the United States but InterMune intends to resubmit the pirfenidone New Drug Application (NDA) to the U.S. FDA early in the third quarter of 2014 to support regulatory registration in the United States.

Regulatory Milestones

• Pirfenidone has been granted Orphan Drug and Fast Track designation by the FDA, and also has been granted Orphan Drug status in Europe.
• On November 4, 2009, InterMune submitted a New Drug Application (NDA) for pirfenidone to the U.S. FDA, seeking approval to market pirfenidone for the treatment of patients with IPF to reduce the rate of decline in lung function. On January 4, 2010, InterMune announced that the FDA had granted Priority Review designation to the pirfenidone NDA. Priority Review designation may be granted by the FDA to an NDA for drugs that have the potential to offer major advances in treatment, or provide a treatment where no adequate therapy exists. Based on the Prescription Drug User Fee Act (PDUFA), the FDA set an action date for the NDA of May 4, 2010. On May 4, 2010, the FDA issued a complete response letter with regard to the pirfenidone NDA, requesting an additional clinical trial to support the efficacy of pirfenidone in IPF. In January 2011 InterMune reported that, as recommended by the FDA in its complete response letter, the company would conduct a new Phase 3 clinical study that would demonstrate a clinically meaningful effect on forced vital capacity. The first patient was enrolled in the study, referred to as the “ASCEND” study, in July of 2011 and enrollment was completed in January 2013. Top-line results from ASCEND were reported in February 2014, as noted above.
• On March 2, 2010, InterMune announced that it had submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA), seeking approval to market pirfenidone for the treatment of IPF patients in the European Union. On March 3, 2011, InterMune announced that the European Commission (EC) had granted marketing authorization for Esbriet^® (pirfenidone). Esbriet is indicated in adults for the treatment of mild to moderate idiopathic pulmonary fibrosis.
• On October 1, 2012, Health Canada approved Esbriet for the treatment of mild to moderate IPF in adult patients. Health Canada designated Esbriet for Priority Review and completed the accelerated review according to target guidelines of 180 days.
• On October 15, 2014, the U.S. FDA approved Esbriet® (pirfenidone) for the treatment of idiopathic pulmonary fibrosis.

Ref:

1. USFDA press release. October 15, 2014 2. official site of Intermune. URL: http://www.intermune.com/pirfenidone