Telitacicept has received the Orphan Drug designation from the US Food and Drug Administration (FDA) for the treatment of myasthenia gravis.

“This is an important step in our quest to address the large unmet need on a global scale for myasthenia gravis,” Jianmin Fang, PhD, the chief executive officer and chief scientific officer of RemeGen, the developers of telitacicept, said in a press release.

Myasthenia gravis is a rare autoimmune disease in which the immune system mistakenly attacks the neuromuscular junction, thereby disrupting the transmission of signals from nerve cells to muscle cells and causing muscle weakness.

Read more about myasthenia gravis

Telitacicept is a novel fusion protein consisting of the extracellular domain of the human transmembrane activator and calcium modulator and cyclophilin ligand interactor (TACI) receptor and the fragment crystallizable domain of human immunoglobulin G. It works by targeting B-cell lymphocyte stimulator (BLyS) and a proliferation-inducing ligand (APRIL), 2 molecules that play a crucial role in the development of B-cells. It is hoped that telitacicept will reduce the B-cell-mediated autoimmune response.

A phase 2 clinical trial testing telitacicept in patients with generalized myasthenia gravis was successfully completed earlier this year.

Orphan Drug designation is granted by the FDA to a drug or biological product that is developed to prevent, diagnose, or treat a rare disease or condition. The designation qualifies sponsors for incentives such as tax credits for certain clinical trials, exemption from user fees, and the potential of 7 years of market exclusivity after approval. A disease is considered rare when it affects less than 200,000 people in the United States.


RemeGen receives Orphan Drug designation from FDA for telitacicept for the treatment of myasthenia gravis. News release. RemeGen Co, Ltd; October 12, 2022.