On October 12, Rongchang biopharmaceutical (Yantai) Co., LTD. (688331.SH/09995.HK) announced that Tercept (R & D code: RC18, trade name: ®) was awarded the qualification of orphan drugs for myasthenia gravis (MG) treatment by the US Food and Drug Administration (FDA). Prior to this, a domestic phase clinical study on the treatment of systemic myasthenia gravis (gMG) had been completed and achieved positive results.
Orphan drugs (Orphan drug), also known as rare disease drugs, refer to the drugs used for the prevention, treatment and diagnosis of rare diseases. The FDA grants orphan drugs as drugs and biologics for rare diseases with fewer than 200,000 people sick in the United States each year, and the recognized drugs can enjoy a rapid track for marketing application, a research and development exclusive period of seven years after marketing, and tax incentives
Myasthenia gravis (MG) is a rare, chronic autoimmune disease, by the postsynaptic membrane acetylcholine receptor, muscle-specific kinase or other acetylcholine receptor-related protein autoantibodies, can cause impaired neuromuscular joint transmission, can affect eye movements, swallowing, speech, activity and breathing, to varying degrees, about 85% of patients will develop symptoms beyond ocular muscle, develop systemic myasthenia gravis (gMG), and even myasthenia gravis, has been included in the first list of rare diseases catalogue in China. At present, the main treatment methods for this disease include cholinesterase inhibitors, glucocorticoids and immunosuppressants, but there are still some patients unable to fully and effectively control the disease due to drug efficacy, tolerability or contraindications to use, and there are a large number of unmet clinical needs.
It is by rongchang biological CEO, chief scientific officer professor Ji'an Jianmin invented to design an antibody fusion protein drug molecules, through the simultaneous inhibition of BLyS and APRIL two cytokines overexpression, "double" prevent the abnormal differentiation and maturity of B cells, thus treating B cell mediated including systemic lupus erythematosus, severe myasthenia gravis, a variety of autoimmune diseases. In March 2021, The first indication was approved by the National Medical Products Administration (NMPA) of China for the treatment of systemic lupus erythematosus (SLE), becoming the world's first dual-target biological new drug in the field of the disease treatment. In the first half of this year, teasept completed a phase clinical trial for myasthenia gravis with positive results.
In addition to systemic lupus erythematosus, myasthenia gravis indications, its multiple other indications in the field of autoimmune disease clinical research has entered / clinical research stage, among them, the optic neuromyelitis spectrum disease, rheumatoid arthritis two indications into clinical stage, multiple sclerosis indication is in domestic clinical, IgA nephropathy, sjogren's syndrome indications have completed domestic clinical, reached the main end point.
