Boehringer Ingelheim has announced that the European Commission (EC) and FDA 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 evaluating nintedanib to understand the disease process and potential benefit of the compound to treat 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 (also known as scleroderma) and up to 90% may develop some degree of lung scarring. In the U.S., it is estimated that SSc-ILD affects up to 86,000 people. SSc-ILD indicates a poor prognosis and accounts for 35% of all disease-related deaths.
"To have nintedanib receive Orphan Drug Designation is an exciting step forward for people living with scleroderma and associated interstitial lung disease, as well as their families," said Robert Riggs, CEO, Scleroderma Foundation.
"This designation represents important progress towards addressing an unmet need and bringing a potential new treatment to those with this rare and devastating disease," said Luke Evnin, Ph.D., chairman, Scleroderma Research Foundation.
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 FDA’s and European Commission’s Orphan Drug Designations for nintedanib are encouraging milestones in our SSc-ILD development program—and underscore our commitment to patients living with rare fibrotic lung diseases,” said Martina Flammer, M.D., vice president, Clinical Development & Medical Affairs Specialty Care, Boehringer Ingelheim Pharmaceuticals. “Building on our positive real-world clinical experience in idiopathic pulmonary fibrosis (IPF), exploring nintedanib’s potential as part of the SENSCIS™ study may revolutionize the way we approach this debilitating disease with unsatisfactory treatment options.”
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 a randomized, double-blind, placebo-controlled study designed to measure the efficacy and safety of nintedanib 150mg 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.
The FDA approved nintedanib, marketed as Ofev, for the treatment of idiopathic pulmonary fibrosis (IPF) on October 15, 2014. Ofev is one of the first FDA-approved drug treatments for IPF and the only kinase inhibitor approved to treat this disease.