Remegen Biologics tetacept was designated as an orphan drug by the US FDA for the treatment of myasthenia gravis

On October 12, Remegen Biopharmaceutical (Yantai) Co.
, Ltd.
(688331.
SH/09995.
HK) announced that Tetacept (R&D code: RC18, trade name: Taiai ^® Obtained orphan drug designation for myasthenia gravis (MG) treatment by the U.
S
.
Food and Drug Administration (FDA).
Prior to this, the domestic phase II clinical study of tetacept in the treatment of systemic myasthenia gravis (gMG) has been completed and obtained positive results
.

Orphan drugs, also known as rare disease drugs, refer to drugs
used to prevent, treat and diagnose rare diseases.
The orphan drug designation granted by the FDA is applicable to drugs and biologics for rare diseases with less than 200,000 cases per year in the United States, and the drugs that have obtained the certification can enjoy incentives such as fast track for marketing application, 7 years of R&D exclusivity after marketing, and tax incentives in the United States
.

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 impaired neuromuscular junction transmission, which can affect eye movement, swallowing, speech, movement, and respiratory function to varying degrees, and about 85% of patients will develop symptoms other than eye muscles, developing generalized myasthenia gravis (gMG), and even myasthenic crisis has been included in China's "First Rare Disease List"
.
At present, the main treatment of the disease includes cholinesterase inhibitors, glucocorticoids and immunosuppressants, but there are still some patients who cannot fully and effectively control the disease due to drug efficacy, tolerance or contraindications to use, and there are a large number of unmet clinical needs
.

Tetacept is an antibody fusion protein drug molecule invented and designed by Professor Fang Jianmin, CEO and Chief Scientific Officer of Remegen, which prevents the abnormal differentiation and maturation of B cells by simultaneously inhibiting the overexpression of BLyS and APRIL cytokines, thereby treating a variety of autoimmune diseases
mediated by B cells, including systemic lupus erythematosus and myasthenia gravis.
In March 2021, the first indication of tatacept was conditionally approved by the National Medical Products Administration (NMPA) in China for the treatment of systemic lupus erythematosus (SLE), becoming the world's first dual-target biological new drug
in the field of treatment of this disease.
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 tetacept in the field of autoimmune diseases has also entered the phase II/III clinical research stage, of which the two indications of neuromyelitis optica spectrum disease and rheumatoid arthritis have entered the phase III clinical stage, the multiple sclerosis indication is undergoing domestic phase II clinical trials, and IgA nephropathy, Sjogren's syndrome and other indications have completed the domestic phase II clinical trial and reached the main endpoint
.

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