Telitacicept Wins Orphan Drug Status to Treat Myasthenia Gravis

The therapy is also being proposed for systemic lupus erythematosus

Marisa Wexler, MS avatar

by Marisa Wexler, MS |

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RemeGen has announced that its experimental therapy Telitacicept has been awarded orphan drug status by the U.S. Food and Drug Administration.

Telitacicept (RC18), an anti-inflammatory therapy developed by RemeGen, has been granted orphan drug designation by the U.S. Food and Drug Administration (FDA) as a potential treatment for myasthenia gravis (MG).

Orphan drug designation, or ODD, is given to therapies that have the potential to treat rare diseases, defined as those affecting fewer than 200,000 people in the U.S. The designation confers certain incentives, including fee waivers, tax credits, and most notably, the potential for seven years of market exclusivity if the therapy ultimately wins FDA approval.

“We’re delighted that the FDA has granted ODD for the RemeGen created proprietary novel fusion protein Telitacicept. This is an important step in our quest to address the large unmet need on a global scale for myasthenia gravis,” Jianmin Fang, CEO and chief scientific officer of RemeGen, said in a press release.

A subsidiary of Yantai Rongchang Pharmaceutical, RemeGen claims telitacicept showed “positive results” in a Phase 2 clinical trial that tested the therapy in people with generalized MG in China.

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MG is caused by an autoimmune attack that interferes with the chemical signals nerve cells use to communicate with muscle cells, ultimately resulting in muscle weakness and other symptoms. The autoimmune attack is driven by immune proteins called antibodies that block nerve-muscle communication by binding to proteins involved in these processes. The most common MG-causing antibodies target a protein called acetylcholine receptor (AChR).

Antibodies, including those that drive MG, are produced by B-cells, a type of immune cell. Telitacicept is designed to reduce the inflammatory activity of B-cells by blocking two proteins that play a key role in their development, BLyS (B-cell lymphocyte stimulator) and APRIL (a proliferation inducing ligand).

The experimental therapy was created by fusing a fragment from an antibody protein with a part of the TACI receptor, a protein involved in regulating B-cell development.

Telitacicept also is being proposed to treat systemic lupus erythematosus (SLE), another autoimmune disease that’s mediated by B-cells. The therapy has been shown to reduce SLE severity in clinical trials and was granted conditional authorization to treat SLE in China last year.