Nintedanib Receives Orphan Drug Designation for Systemic Sclerosis

Date: Wed 28th September 2016

Published by Pharmacy Times: September 27, 2016.


The FDA has granted Orphan Drug Designation to Boehringer Ingelheim's nintedanib for the treatment of systemic sclerosis (SSc), also known as scleroderma, including associated interstitial lung disease (SSc-ILD).

“Scleroderma and associated interstitial lung disease have a devastating impact on the patient community, and we welcome this important news [about] a potential new treatment,” said Joep Welling, of the Federation of European Scleroderma Associations, in a press release. “It's a crucial step forward in helping to address an unmet need and represents important progress for patients with this rare disease.”

SSc is a disfiguring, disabling, and potentially fatal rare disease that can cause scarring of the skin, lungs, and other organs. An estimated 2 million individuals worldwide have SSc, 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.

Nintedanib's safety and efficacy in treating SSc is being evaluated in SENSCIS, the largest trial to date in this disease area. The randomized, double-blind, placebo-controlled study is designed to measure the nintedanib 150 mg taken twice daily over 52 weeks up to 100 weeks in patients with SSc-ILD. The primary endpoint is the annual rate of decline in forced vital capacity, while secondary endpoints include the absolute change from baseline in the modified Rodnan Skin Score and the Saint George´s Respiratory Questionnaire total score.

Nintedanib (Ofev) is already approved for the treatment for idiopathic pulmonary fibrosis, a rare lung disease. It has been shown to slow the progression of the disease, as measured by the annual rate of decline in lung function.