The European Commission (EC) and the U.S. Food and Drug Administration (FDA) have both designed Ofev (nintedanib; manufactured by Boehringer Ingelheim) an Orphan Drug (ODD) for the treatment of scleroderma, as well as the associated interstitial lung disease. A large Phase 3 trial evaluating its effectiveness in scleroderma is now recruiting patients.
The EC grants ODD for therapeutic agents aiming to treat a life-threatening or chronically debilitating condition affecting no more than five in 10,000 people, and for which there is no or only unsatisfactory treatment options. The FDA grants the status to investigational compounds intended to effectively and safely diagnose, prevent and treat rare diseases affecting less than 200,000 people in the U.S. The designation provides a number of advantages to a drug’s maker to speed its clinical testing and development.
“The FDA’s and European Commission’s Orphan Drug Designations for nintedanib are encouraging milestones … and underscore our commitment to patients living with rare fibrotic lung diseases,” said Dr. Martina Flammer, vice president of Clinical Development & Medical Affairs Specialty Care at Boehringer Ingelheim Pharmaceuticals, in a press release issued by the company.
The news was also positively received by scleroderma charities.
“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, chief executive officer of the Scleroderma Foundation.
Dr. Luke Evnin, chairman of Scleroderma Research Foundation, added: “This designation represents important progress towards addressing an unmet need and bringing a potential new treatment to those with this rare and devastating disease.”
Ofev is a kinase inhibitor, which has already been approved for the FDA for the treatment of idiopathic pulmonary fibrosis. The safety and efficacy of the drug as a scleroderma treatment is currently being tested in a Phase 3 clinical trial called SENSCIS (safety and efficacy of nintedanib in systemic sclerosis).
SENSCIS (NCT02597933) is a randomized, double-blind, placebo-controlled trial to assess Ofev in people with scleroderma at a dose of 150 mg twice a day over 52 weeks. The researchers will analyze how the drug affects disease progression by measuring the annual rate of lung function decline through forced vital capacity (FVC). The team will also assess the impact of the treatment by measuring skin thickness and evaluating participants’ health-related quality of life.
The trial is currently recruiting patients at 179 locations across the U.S., Europe and Canada, and in India, Japan, and elsewhere. For more information and how to participate, please visit this link.