Ra Pharmaceuticals issued the following announcement on Sep. 4.
Ra Pharmaceuticals, Inc. (Nasdaq:RARX) today announced that the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation to zilucoplan for the treatment of myasthenia gravis. Ra Pharma is developing zilucoplan, a self-administered macrocyclic peptide inhibitor of complement component 5 (C5), for the treatment of generalized myasthenia gravis (gMG) and other rare, tissue-based complement-mediated diseases.
“gMG is a chronic and debilitating neuromuscular disease that affects more than 60,000 patients in the U.S. who have limited treatment options,” said Doug Treco, Ph.D., President and Chief Executive Officer of Ra Pharma. “We’ve designed zilucoplan, a macrocyclic peptide inhibitor of C5, as an easy-to-use, self-administered subcutaneous treatment option to address the underlying cause of gMG through targeted complement control. With site activations underway, we are on track to initiate our single, pivotal, 12-week, Phase 3 trial of zilucoplan for the treatment of gMG in the second half of this year.”
About Orphan Drug Designation
The U.S. Food and Drug Administration (FDA) grants Orphan Drug Designation status to products that treat rare diseases, providing incentives to sponsors developing drugs or biologics. The FDA defines rare diseases as those affecting fewer than 200,000 people in the U.S. at the time of designation. Orphan Drug Designation qualifies the sponsor for certain development incentives, including tax credits for qualified clinical testing.
About Myasthenia Gravis
Myasthenia gravis (MG) is a chronic, autoimmune, neuromuscular disease characterized by weakness and fatigue of skeletal muscles. Patients with MG present with muscle weakness that becomes increasingly severe with repeated use and recovers with rest. Weakness can be localized to specific muscles, such as those responsible for eye movements, but often progresses to affect a broader range, including head, limb, and respiratory muscles. This progression is often described as the generalized, or severe, form of the disease. gMG is estimated to affect approximately 60,000 people in the U.S. alone.
About Zilucoplan
Ra Pharma is developing zilucoplan and zilucoplan extended release (XR) for generalized myasthenia gravis (gMG), immune-mediated necrotizing myopathy (IMNM), and other tissue-based complement-mediated disorders with high unmet medical need. The product candidate is designed for convenient subcutaneous (SC) self-administration. Zilucoplan is an investigational, synthetic, macrocyclic peptide discovered using Ra Pharma's powerful proprietary drug discovery technology. The peptide is designed to bind complement component 5 (C5) with sub-nanomolar affinity and allosterically inhibit its cleavage into C5a and C5b upon activation of the classical, alternative, or lectin pathways.
About Ra Pharmaceuticals, Inc.
Ra Pharmaceuticals is a clinical-stage biopharmaceutical company focused on leading the field of complement biology to bring innovative and accessible therapies to patients with rare diseases. The Company discovers and develops peptides and small molecules to target key components of the complement cascade. For more information, please visit: www.rapharma.com.
Original source can be found here.
