Ra Pharmaceuticals Inc. said the U.S. Food and Drug Administration granted the orphan-drug status to its medicine zilucoplan for treating a type of chronic neuromuscular disease called generalized myasthenia gravis.
Myasthenia gravis, or MG, is an autoimmune disease characterized by weakness and fatigue of skeletal muscles responsible for breathing and moving body parts and affects about 60,000 people in the U.S.
The Cambridge, Mass.-based company is developing zilucoplan as a self-administered treatment containing complement component 5, or C5, which is a type of protein that plays an important role in inflammatory and cell killing processes. The company plans to study the medicine in a phase 3 trial later this year.
The FDA awards orphan-drug designations to medicines that treat diseases affecting fewer than 200,000 people in the country. The designation allows for several key benefits and incentives, including grant funding for clinical trial costs, tax credits, user fee waivers and the potential for a seven-year period of drug marketing exclusivity upon approval.
