Boehringer Ingelheim’s investigational therapy nintedanib* receives the first FDA breakthrough therapy designation in IPF

Boehringer Ingelheim announced that for the first time the U.S. Food & Drug Administration (FDA) has granted Breakthrough Therapy designation for a treatment in idiopathic pulmonary fibrosis (IPF), a devastating and fatal lung disease. Nintedanib is an investigational therapy currently under FDA and European Medicines Agency (EMA review) for IPF.

“We’re excited nintedanib* has been granted breakthrough therapy designation in the US, which we hope will make the treatment available to IPF patients as quickly as possible. Currently there are no FDA-approved treatments available for IPF. We are committed to working with all regulatory bodies to make nintedanib* available to people living with this serious and life-threatening lung disease.” said Professor Klaus Dugi, Chief Medical Officer, Boehringer Ingelheim.

The Breakthrough Therapy designation process was established by the FDA in 2012. It is intended to facilitate and expedite the development and review of treatments for serious or life-threatening conditions if preliminary clinical evidence indicates that the therapy may demonstrate substantial improvement over existing therapies on one or more clinically significant endpoints.¹

IPF is a debilitating and fatal lung disease that causes permanent scarring of the lungs, difficulty breathing and decreases the amount of oxygen the lungs can supply to the major organs of the body.² IPF affects as many as 14-43 people per 100,000 worldwide.³

Results from two global Phase III studies (INPULSIS™-1 and INPULSIS™-2) evaluating the efficacy and safety of nintedanib* for the treatment of IPF were presented at the American Thoracic Society (ATS) International Conference and published in the New England Journal of Medicine in May 2014.⁴

Nintedanib* is the first targeted treatment for IPF that has consistently demonstrated to slow disease progression in IPF by significantly reducing the annual decline in lung function by approximately 50%.⁴

This effect on disease progression was further supported in the pooled data set by a positive signal in reducing the risk of acute exacerbations by 38% (p=0.08) and a significant risk reduction in confirmed or suspected acute exacerbation by 68% (p=0.005).⁴

*Nintedanib is an investigational compound. Its safety and efficacy have not yet been fully established.

About nintedanib*Nintedanib is a small molecule tyrosine kinase inhibitor (TKI) in development by Boehringer Ingelheim for idiopathic pulmonary fibrosis (IPF).⁵ It targets growth factor receptors, which have been shown to be potentially involved in pathomechanisms of pulmonary fibrosis, most importantly the platelet-derived growth factor receptor (PDGFR), fibroblast growth factor receptor (FGFR) and vascular endothelial growth factor receptor (VEGFR). ^6,7 By blocking the signaling pathways that are involved in fibrotic processes, it is believed that nintedanib* has the potential to reduce disease progression in IPF by slowing the decline of lung function.^7,8 Nintedanib* is also in clinical development as a treatment option for cancer, including non-small cell lung cancer, ovarian cancer, colorectal cancer and hepatocellular carcinoma.

About idiopathic pulmonary fibrosis

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About Boehringer IngelheimThe Boehringer Ingelheim group is one of the world’s 20 leading pharmaceutical companies. Headquartered in Ingelheim, Germany, Boehringer Ingelheim operates globally with 142 affiliates and a total of more than 47,400 employees. The focus of the family-owned company, founded in 1885, is researching, developing, manufacturing and marketing new medications of high therapeutic value for human and veterinary medicine.

Taking social responsibility is an important element of the corporate culture at Boehringer Ingelheim. This includes worldwide involvement in social projects, such as the initiative “Making more Health” and caring for the employees. Respect, equal opportunities and reconciling career and family form the foundation of the mutual cooperation. In everything it does, the company focuses on environmental protection and sustainability.

In 2013, Boehringer Ingelheim achieved net sales of about €14.1 billion. R&D expenditure corresponds to 19.5% of its net sales.

References

¹ U.S. Food and Drug Administration/Center for Drug Evaluation and Research/Center for Biologics Evaluation and Research. (2014) Guidance for Industry: Expedited Programs for Serious Conditions - Drugs and Biologics. Silver Spring, MD.
² Raghu G., et al. An Official ATS/ERS/JRS/ALAT Statement: Idiopathic Pulmonary Fibrosis: Evidence-based Guidelines for Diagnosis and Management. American Journal of Respiratory and Critical Care Medicine. 2011; 183: 788–824, 2011.
³ Raghu G., et al. Incidence and prevalence of idiopathic pulmonary fibrosis. Am J Respir Crit Care Med. 2006; 174:810-816
⁴ Richeldi, L., et al. Efficacy and Safety of Nintedanib in Idiopathic Pulmonary Fibrosis. New England Journal of Medicine. 2014, Vol. 370, pp.2071-2082. Published online on May 18, 2014.
⁵Richeldi L., et al. Efficacy of a tyrosine kinase inhibitor in idiopathic pulmonary fibrosis. N Engl J Med. 2011;365:1079-1087.
⁶Selman M, et al. Idiopathic pulmonary fibrosis: prevailing and evolving hypotheses about its pathogenesis and implications for therapy. Ann Intern Med. 2001;134:136-51.
⁷Hilberg F., et al. BIBF 1120: triple angiokinase inhibitor with sustained receptor blockade and good antitumor efficacy. Cancer Res. 2008;68:4774-4782.
⁸Wollin L., et al. Antifibrotic and Anti-inflammatory Activity of the Tyrosine Kinase Inhibitor Nintedanib in Experimental Models of Lung Fibrosis. J Pharmacol Exp Ther 2014;349:209–220.