Old person hospital admissionThe European Commission (EC) and US Food and Drug Administration (FDA) have granted Orphan Drug Designation to nintedanib for the treatment of systemic sclerosis (SSc), including the associated interstitial lung disease (SSc-ILD).

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. Worldwide, an estimated two million people have systemic sclerosis and up to 90% may develop some degree of lung scarring. SSc-ILD indicates a poor prognosis and accounts for 35% of all disease-related deaths.

“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 US, 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 SENSCISTM 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.”

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.