InterMune Snags FDA Breakthrough Status For IPF Drug

The U.S. Food and Drug Administration (FDA) has designated Breakthrough Therapy status to biotech firm InterMune’s pirfenidone as an investigational treatment for adult patients with idiopathic pulmonary fibrosis (IPF).

Pirfenidone is an investigational orally active, anti-fibrotic agent that works to inhibit the synthesis of TGF-beta, a chemical mediator responsible for several cell functions including proliferation and differentiation, and it has been implicated in fibrosis. The drug has secured marketing approval under the brand name Esbriet in the EU for the treatment of mild to moderate IPF. In 2012, InterMune received approval from Health Canada for Esbriet following its Priority Review.

Dan Welch, Chairman, CEO and President of InterMune, said, “The Breakthrough Therapy Designation underscores the significant need to help patients with this irreversible and ultimately fatal disease, particularly as no FDA-approved therapies are currently available. We are pleased that the FDA recognized the importance of pirfenidone as a potential new therapy for IPF, a disease with great unmet medical need.”

Earlier this year, the company presented the data from the Phase III ASCEND study of pirfenidone in IPF patients at the American Thoracic Society (ATS) and subsequently published it in the New England Journal of Medicine.

Idiopathic pulmonary fibrosis (IPF) is an irreversible, ultimately fatal disease marked by increasing loss of lung function due to scarring or fibrosis in the lungs. Fibrosis lessens the ability of the lungs to absorb oxygen over time. IPF represents an area of significant unmet clinical need with a low rate of survival (5-year survival rate of around 20 to 40 percent), which makes the disease more rapidly lethal than breast, colorectal, and ovarian cancer.

The FDA’s Breakthrough Therapy is reserved for therapies and drugs that are designed to treat a life threatening disease or a serious condition. The candidate treatments may also offer significant improvement over current therapies on one or more clinically meaningful endpoints. The company resubmitted its New Drug Application (NDA) for pirfenidone in May and announced a target FDA review date of six months following submission.