ADC Reviews

Tetacept Receives FDA Orphan Drug Designation for Myasthenia Gravis (MG)

Myasthenia gravis

On October 12, Rongchang Bio announced that Taitacept (R&D code: RC18, trade name: Taiai®) was granted orphan drug designation by the U.S. Food and Drug Administration (FDA) for the treatment of myasthenia gravis (MG) identified. Prior to this, the domestic phase II clinical study of tetacept in the treatment of generalized myasthenia gravis (gMG) has been completed, and positive results have been obtained.

Orphan drugs, also known as rare disease drugs, refer to drugs used for the prevention, treatment and diagnosis of rare diseases. The orphan drug designation granted by the FDA is applicable to drugs and biologics for rare diseases that affect less than 200,000 people in the United States each year. The approved drugs can enjoy fast-track marketing applications in the United States, a 7-year R&D exclusivity period after marketing, and Tax incentives and other incentives.

Myasthenia gravis (MG) is a rare, chronic autoimmune disease caused by autoantibodies against acetylcholine receptors, muscle-specific kinases, or other acetylcholine receptor-related proteins on the postsynaptic membrane, resulting in the neuromuscular junction Impaired transmission can affect eye movement, swallowing, speech, activity and respiratory function to varying degrees. About 85% of patients will develop symptoms other than the eye muscles, develop generalized myasthenia gravis (gMG), and even develop myasthenic crisis. , has been included in my country’s “First Batch of Rare Disease List”. At present, the main treatment methods for this disease include cholinesterase inhibitors, glucocorticoids and immunosuppressants, but there are still some patients who cannot fully and effectively control the disease due to problems such as drug efficacy, tolerance or contraindications, and there are a large number of unresolved diseases. met clinical needs.

Taitacept is an antibody fusion protein drug molecule invented and designed by Professor Fang Jianmin, CEO and Chief Scientific Officer of Rongchang Bio, by simultaneously inhibiting the overexpression of two cytokines, BLyS and APRIL. Mature to treat B cell-mediated autoimmune diseases including systemic lupus erythematosus, myasthenia gravis indications. In March 2021, the first indication of Taitacept was approved by the National Medical Products Administration (NMPA) in China for the treatment of systemic lupus erythematosus (SLE), becoming the world’s first in the field of treatment of this disease. Dual-target biological drugs. In the first half of this year, Tetacept completed a Phase II clinical trial for the treatment of myasthenia gravis, and achieved positive results.

In addition to the indications for systemic lupus erythematosus and myasthenia gravis, the clinical research of tacitcept for other indications in the field of autoimmune diseases has also entered the phase II/III clinical research stage. Among them, neuromyelitis optica spectrum disorders, Two indications for rheumatoid arthritis have entered the phase III clinical stage, multiple sclerosis indications are undergoing domestic phase II clinical trials, and indications such as IgA nephropathy and Sjögren’s syndrome have completed domestic phase II clinical trials, reaching the primary endpoint.