China-based pharma firm RemeGen (HKG: 9995, SHA: 688331) has announced receiving Orphan Drug Designation (ODD) status from the US FDA for its fusion protein drug telitacicept in myasthenia gravis (MG). This designation highlights the potential of telitacicept in treating this rare and chronic autoimmune disease. The drug previously concluded a Phase II clinical study in generalized myasthenia gravis (gMG) in China, with positive results.
Myasthenia Gravis: Disease Overview
Myasthenia gravis is a rare and chronic autoimmune disease caused by the dysfunction of nerve muscle junction transmission. It is characterized by a long course, difficulty in cure, and a high likelihood of relapse among patients. Current treatments, including cholinesterase inhibitors, glucocorticoids, and immunosuppressants, often provide unsatisfactory clinical outcomes over long-term use.
Telitacicept: Drug Profile and Development
Telitacicept is a novel recombinant fusion protein targeting both B lymphocyte stimulator (BLyS) and a proliferating-inducing ligand (APRIL). The drug was conditionally approved in China to treat systemic lupus erythematosus (SLE) in March 2021, becoming the first domestically developed SLE drug in the country. RemeGen set a standard retail price of RMB 2,586 (USD 359) per shot for telitacicept on March 31, the day it was officially launched into the market. The drug was included in the National Reimbursement Drug List (NRDL) at the end of 2021.
Clinical Studies and Future Outlook
In addition to SLE, telitacicept is undergoing Phase II/III clinical studies in several indications, including Sjogren’s syndrome, IgA nephropathy, neuromyelitis optica, multiple sclerosis, and myasthenia gravis. The Orphan Drug Designation from the FDA underscores the potential of telitacicept to address significant unmet medical needs in rare diseases like myasthenia gravis.-Fineline Info & Tech
