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RemeGen Receives Orphan Drug Designation from FDA for Telitacicept for the Treatment of Myasthenia Gravis

This will address the large unmet need on a global scale for Myasthenia Gravis.

RemeGen Co., Ltd., a commercial-stage biotechnology company, announced that the U.S. Food and Drug Administration (FDA) has granted orphan-drug designation (ODD) for its proprietary novel fusion protein Telitacicept (RC18) for the treatment of myasthenia gravis (MG).

Myasthenia gravis (MG) is a chronic autoimmune neuromuscular disease that causes patients to be prone to fluctuating muscle weakness. It can affect eye movements, swallowing, speech, and respiratory functions to varying degrees and can even be life-threatening in severe cases. The disease, although rare, is a long-term condition that currently is difficult to cure. In 2018, it was added to the National Rare Diseases Registry System of China, the first national list of rare diseases to be issued in the country.

At present, the most common treatment options to alleviate symptoms for MG include cholinesterase inhibitors, glucocorticoids, immunosuppressants, intravenous immunoglobulin, plasma exchange, anti-CD20 monoclonal antibodies (mAbs), and thymectomy. These treatments can have certain side effects, while fast-acting treatments such as plasma replacement may have an impact on the immune system due to the removal of other macromolecules and poor accessibility. Therefore, a large unmet clinical need is currently being addressed by RemeGen.

“We’re delighted that the FDA has granted ODD for the RemeGencreated proprietary novel fusion protein Telitacicept,” said Dr. Jianmin Fang, CEO and Chief Scientific Officer of RemeGen. “This is an important step in our quest to address the large unmet need on a global scale for myasthenia gravis, and follows on from our successful Phase II clinical study on patients in China earlier this year.

In the first quarter of this year, the company announced the completion of a Phase II clinical study of Telitacicept for the treatment of generalised myasthenia gravis (gMG) in Chinese patients, which showed positive results. [APBN]

Source: RemeGen Co., Ltd.