Ra Pharmaceuticals Receives Orphan Drug Designation from the U.S. FDA for Zilucoplan for the Treatment of Myasthenia Gravis
“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
About Orphan Drug Designation
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.
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.
This press release contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, including, but not limited to, statements regarding Ra Pharma's ability to expand patient access to important therapies, the potential, safety, efficacy, and regulatory and clinical progress of Ra Pharma's product candidates, including without limitation zilucoplan and zilucoplan XR, beliefs regarding clinical trial data, and statements regarding trial design, timeline, and enrollment of Ra Pharma's ongoing and planned clinical programs, including without limitation the Phase 3 trial of zilucoplan for the treatment of gMG. All such forward-looking statements are based on management’s current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include the risks that Ra Pharma's product candidates, including zilucoplan and zilucoplan XR, will not successfully be developed or commercialized, in the timeframe we expect or at all; the risk that Ra Pharma may fail to enroll patients in its clinical trials, which may cause delays or other adverse effects; the risk that Ra Pharma may be unable to obtain orphan drug designation or to maintain the benefits associated with orphan drug status, including market exclusivity; as well as the other factors discussed in the “Risk Factors” section in Ra Pharma’s most recently filed Annual Report on Form 10-K, as well as other risks detailed in Ra Pharma’s subsequent filings with the