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FDA Breakthrough Therapy Designation: 2 Drugs Approved For Idiopathic Pulmonary Fibrosis

October 15, 2014

The FDA announces on October 15th the approval of two Breakthrough Therapy Designations (BTDs) for the treatment of the rare disease,  Idiopathic Pulmonary Fibrosis (IPF):

•   InterMune’s Esbriet (Pirfenidone)

•   Boehringer Ingelheim Pharmaceuticals’ Ofev (Nintedanib).

I – What is IPF ?

IPF is a chronic, progressive, severely debilitating and ultimately fatal lung disease – characterized by progressive permanent scarring of lung tissue. There are approximately 100,000 Americans with IPF. Up until today, there were no currently FDA-approved treatment options.

II – Esbriet (Pirfenidone)

InterMune, a global biotechnology company focusing on the development and commercialization of therapies in pulmonology and orphan fibrotic diseases, announces in July 2014 that the company receives the FDA BTD for orphan drug Esbriet (Pirfenidone), an oral drug for adult patients with IPF. The FDA grants Esbriet Fast Track, Priority Review, Orphan Drug Designation, and Breakthrough Therapy Designation. Esbriet is approved ahead of its PDUFA Date of November 23, 2014, the date the FDA was scheduled to complete the review of the drug application.

Esbriet was developed by InterMune, which was acquired by Roche in August 2014 for $8.3 billion.

According to a Press Release today, Esbriet will be available to patients in the United States within 2 weeks, along with a comprehensive patient support program designed to help with education, access, and financial support.

The global regulatory history of Esbriet is interesting, as the drug has been on the global market for a few years, but was not approved in the US before today’s approval. InterMune resubmits its Pirfenidone New Drug Application (NDA) to the FDA in May 2014, in response to a Complete Response Letter (CRL) the company receives in May 2010. In the CRL, the FDA recommends an additional Phase III clinical trial to support the efficacy of Pirfenidone. InterMune conducts the Phase III ASCEND clinical trial and results are presented at the May 2014 meeting of the American Thoracic Society and is also published on-line in the New England Journal of Medicine.

The European Commission (EC) grants marketing authorization in February 2011, in all 28 EU member states, for Esbriet for the treatment of adults with mild to moderate IPF. Esbriet also receives approval for marketing in Iceland and Norway. Commercial sales of Esbriet in the EU include the key markets of Germany, France, Italy, and the UK.

Other global markets include:

•   2008 launch of Pirespa (Pirfenidone) in Japan

•   2012 launch of Pirespa in South Korea

•   January 2013 Esbriet launch in Canada

•   Approval in China, India, Argentia, and Mexico.

III – Ofev (Nintedanib)

Boehringer Ingelheim (BI) announces in July 2014, that the company’s investigational orphan drug Ofev (Nintedanib) receives the FDA BTD for the treatment of IPF. Similar to Esbriet, the FDA grants Ofevt Fast Track, Priority Review, Orphan Drug Designation, and Breakthrough Therapy Designation. Ofev is being approved ahead of its PDUFA date of January 2, 2015, the date the agency was scheduled to complete the review of the drug application.

According to today’s BI Press Release, Ofev will be available to the IPF community within 10 days, along with comprehensive patient support resources.

Other Ofev Global Regulatory Activity

Regulatory Date Regulatory Agency & Action Taken
June 2011 FDA Orphan Drug Designation
April 2013 European Commission (EC) Orphan Designation
June 2013 FDA Fast Track Designation
June 2014 FDA Priority Review Designation
June 2014 EMA validates application for marketing authorization & grants accelerated review
July 2014 FDA BTD
October 2014 FDA Approval

.

IV – US IPF Market Race And Availability

According to a Press Release today, Esbriet will be available to patients in the United States within 2 weeks, along with a comprehensive patient support program designed to help with education, access, and financial support.

According to today’s BI Press Release, Ofev will be available to the IPF community within 10 days, along with comprehensive patient support resources.

According to an online San Francisco Business Times article, Esbriet will cost about $94,000/year/patient in the United States and about $40,000 in Europe and Canada, though the price varies from country to country. No pricing for Ofev as of this Blog Post has been made.

In an online 2013 PharmaTimes article, the following data is presented from GlobalData on the IPF global market :

•   Expects IPF therapy sales across US, France, Germany, Italy, Spain, and the UK to increase dramatically from $49 million in 2012 to over $1.1 billion by 2017

•   US IPF therapy sales is expected to increase from a value of $6.5 million in 2012 to $696 million in 2017

•   European IPF therapy sales is expected to increase from $43 million in 2012 to $419 million in 2017

•   The anticipated launch in the US of InterMune’s Esbriet and BI’s Ofev, in a market that currently has no therapeutic options, will cause the US market to exponentially grow to about $500 million in 2015.

It will be interesting to watch over the next year the neck-to-neck race between InterMune’s Esbriet and BI’s Ofev, to see which product will launch first in the US market, what strategies both companies will use, and what market share each IPF drug will take.

References

FDA BTD Chart

FDA BTD Approval Chart

FDA BTD Statistics Chart.

Please Note: “Kalymnos 2005 022” by David Bolius [CC By-SA 2.5] | Wikimedia Commons.

Copyright © 2012-2014, Orphan Druganaut Blog. All rights reserved.

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