The US Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to InterMune, Inc's pirfenidone, an orally active, anti-fibrotic agent. This designation is reserved for drugs that are intended to treat a serious or life threatening disease or condition and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over existing therapies on one or more clinically significant endpoints.  

As described in the FDA Fact Sheet: Breakthrough Therapies, "If a drug is designated as breakthrough therapy, FDA will expedite the development and review of such drug." In May, InterMune resubmitted its New Drug Application (NDA) for pirfenidone and noted a target FDA review of six months under the Prescription Drug User Fee Act.  Pirfenidone is an investigational treatment for adult patients with idiopathic pulmonary fibrosis (IPF).

"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," said Dan Welch, chairman, chief executive officer and president of InterMune.  "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."

Pirfenidone is an orally active, anti-fibrotic agent that inhibits the synthesis of TGF-beta, a chemical mediator that controls many cell functions including proliferation and differentiation, and plays a key role in fibrosis.  Pirfenidone also inhibits the synthesis of TNF-alpha, a cytokine that is known to have an active role in inflammation.

On February 28, 2011, the European Commission (EC) granted marketing authorization for Esbriet (pirfenidone) for the treatment of adults with mild to moderate IPF.  The approval authorized marketing of Esbriet in all 28 EU member states.  Esbriet has since been approved for marketing in Norway and Iceland. In 2011, InterMune launched commercial sales of pirfenidone in Germany under the trade name Esbriet, and Esbriet is now also commercially available in various European countries, including key markets such as France, Italy and the UK.

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.  InterMune launched Esbriet in Canada in January 2013.

Pirfenidone has been marketed as Pirespa since 2008 in Japan and since 2012 in South Korea by Shionogi & Co. Ltd.  Under different trade names, pirfenidone is also approved for the treatment of IPF in China, India, Argentina and Mexico.

Pirfenidone is not approved for sale in the United States.

Idiopathic pulmonary fibrosis (IPF) is an irreversible and ultimately fatal disease characterized by progressive loss of lung function due to fibrosis (scarring) in the lungs, which hinders the ability of lungs to absorb oxygen.  IPF inevitably causes shortness of breath, and a deterioration in lung function and exercise tolerance.  IPF patients follow different and unpredictable clinical courses and it is not possible to predict if a patient will progress slowly or rapidly, or when the rate of decline may change.  Periods of transient clinical stability in IPF, when they occur, inevitably give way to continued disease progression.  The median survival time from diagnosis is two to five years, with a five-year survival rate of approximately 20-40 percent, which makes IPF more rapidly lethal than many malignancies, including breast, ovarian and colorectal cancers.  IPF typically occurs in patients over the age of 45, and tends to affect slightly more men than women.

InterMune is a biotechnology company focused on the research, development and commercialization of innovative therapies in pulmonology and orphan fibrotic diseases.  In pulmonology, the company is focused on therapies for the treatment of idiopathic pulmonary fibrosis (IPF), a progressive, irreversible, unpredictable and ultimately fatal lung disease.