Orphan Drug Designation Granted to Nintedanib for Treatment of Systemic Sclerosis, including Associated Interstitial Lung Disease

Ingelheim, Germany, September 9, 2016 –  Boehringer Ingelheim today announced that the European Commission (EC) and U.S. Food and Drug Administration (FDA) have granted Orphan Drug Designation to nintedanib for the treatment of systemic sclerosis (SSc, also known as scleroderma), including the associated interstitial lung disease (SSc-ILD). 

SENSCIS^™, the largest trial to date in this disease area, is specifically evaluating nintedanib to understand the disease process and potential benefit of the compound to treat SSc-ILD.¹

Systemic sclerosis, commonly referred to as “scleroderma,” is a disfiguring, disabling and potentially fatal rare disease that can cause scarring of the skin, lungs (SSc-ILD) and other organs.^2,3 Worldwide, an estimated two million people have systemic sclerosis³ (also known as scleroderma) and up to 90% may develop some degree of lung scarring.² SSc-ILD indicates a poor prognosis and accounts for 35 percent of all disease-related deaths.⁴

“Scleroderma and associated interstitial lung disease have a devastating impact on the patient community, and we welcome this important news that a potential new treatment has received Orphan Drug Designation. It’s a crucial step forward in helping to address an unmet need and represents important progress for patients with this rare disease.” said Joep Welling, Federation of European Scleroderma Associations (FESCA aisbl).

Orphan Drug Designation is generally granted by the EC for a therapeutic agent intended to treat a life-threatening or chronically debilitating disease that affects no more than five people in 10,000, and for which there is no or only unsatisfactory treatment options or the medicine will be of significant benefit to those affected by that condition. In the U.S., the FDA grants the status to investigational compounds intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people. 

 “The Orphan Drug Designation granted by both the European Commission and FDA for nintedanib is an important milestone in the development of much needed therapies for the various complications of systemic sclerosis also known as scleroderma,” said Dr William Mezzanotte, Therapeutic Area Head, Respiratory Medicine at Boehringer Ingelheim. “The need for treatment options for people with SSc-ILD is significant, and the SENSCIS^TM trial is an important first step in exploring the benefit of nintedanib in addressing the complications of systemic sclerosis.  The success of nintedanib in treating IPF, in both clinical trials and the clinical world, along with similarities of IPF to other fibrotic lung diseases, including SSc-ILD, gives us great hope that this important medicine can help patients with SSc-ILD. At Boehringer Ingelheim, we are committed to transforming fibrotic lung diseases from fatal diseases to chronic, treatable ones.”

Notes to Editor

About SENSCIS^™ and nintedanib
The SENSCIS^™ (Safety and Efficacy of Nintedanib in Systemic SClerosIS) study has the potential to provide advances in the treatment of this devastating condition and to help address the unmet need for the many people living with systemic sclerosis or SSc-ILD. Nintedanib is currently being evaluated in SENSCIS^™, a randomized, double-blind, placebo-controlled study designed to measure the efficacy and safety of nintedanib 150 mg twice daily over 52 weeks up to a maximum of 100 weeks in people with SSc-ILD. The primary endpoint is the annual rate of decline in forced vital capacity (FVC), a measure of disease progression. Key secondary endpoints include the absolute change from baseline in the modified Rodnan Skin Score (mRSS), which is an evaluation of people’s skin thickness, and the absolute change from baseline in the Saint George´s Respiratory Questionnaire (SGRQ) total score, which measures the health-related quality of life of people with lung diseases to assess the impact of treatment.¹

Additional information is also available at: clinicaltrials.gov/ct2/show/NCT02597933.

Nintedanib, marketed as OFEV^®, is approved for the treatment of the rare lung disease IPF (Idiopathic Pulmonary Fibrosis) and has been shown to slow disease progression as measured by annual rate of decline in lung function.⁵ 

About Systemic Sclerosis and Interstitial Lung Disease
Systemic sclerosis (SSc, also known as scleroderma) is a rare and serious disease characterized by thickening and scarring of connective tissue of multiple organs in the body.^2,3 It is difficult to diagnose because a lot of organ systems are involved and its symptoms are similar to other autoimmune diseases.⁶ The disease impacts four times as many women as men, and the onset of the disease typically occurs at a young age – between 35 and 55 years.^6,7

Nearly all people with systemic sclerosis have hardening and thickening of patches of skin. The disease also can cause scarring in the lungs, heart, and kidneys, which can be debilitating and may become life-threatening. ^2,3

When the disease affects the lungs, ultimately resulting in scarring, it is called SSc-ILD.² Shortness of breath and difficulty in performing daily activities are common symptoms of SSc-ILD.⁸ Lung involvement, like ILD, is the leading cause of death among people with systemic sclerosis.⁹

For more information, please visit https://www.boehringer-ingelheim.com/respiratory/ssc-ild

About Boehringer Ingelheim
Boehringer Ingelheim is one of the world’s 20 leading pharmaceutical companies. Headquartered in Ingelheim, Germany, Boehringer Ingelheim operates globally through 145 affiliates and a total of some 47,500 employees. The focus of the family-owned company, founded in 1885, is on researching, developing, manufacturing and marketing new medications of high therapeutic value for human and veterinary medicine.

Social responsibility is an important element of the corporate culture at Boehringer Ingelheim. This includes worldwide involvement in social projects through, for example, the initiative “Making More Health” while also caring for employees. Respect, equal opportunity and reconciling career and family form the foundation of mutual cooperation. The company also focuses on environmental protection and sustainability in everything it does.

In 2015, Boehringer Ingelheim achieved net sales of about 14.8 billion euros. R&D expenditure corresponds to 20.3 per cent of net sales.

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