RemeGen Co., Ltd announced on October 27, 2024, that China's National Medical Products Administration (NMPA) Center for Drug Evaluation has officially accepted the Biologics License Application (BLA) for Telitacicept (RC18, trade name: Tai'ai®) to treat generalized myasthenia gravis (gMG) and granted priority review. The innovative dual-target fusion protein has already demonstrated success in a Phase III clinical trial that achieved its primary endpoint.
Clinical Trial Success Drives Regulatory Milestone
The Phase III clinical trial was a multicenter, randomized, double-blind, placebo-controlled study conducted in China with a Beijing hospital as the leading site. Eligible patients were randomized to receive either Telitacicept 240 mg or placebo in a 1:1 ratio. The results demonstrated that Telitacicept had favorable efficacy and safety profiles, evidenced by the continuous reduction of clinical symptoms among gMG patients in the treatment group.
RemeGen announced the achievement of the trial's primary endpoint on August 13, 2024, leading to the company's plan to file the BLA submission that has now received priority review status.
Addressing Significant Unmet Medical Need
Myasthenia gravis is a rare chronic autoimmune disease that has been included in China's First Batch of Rare Disease Catalog. According to a report by Frost & Sullivan, approximately 1.146 million individuals worldwide are predicted to be affected by MG as of 2025, with around 217,000 in China.
The disease arises from immunoglobulin G (IgG) impairing synaptic transmission at the neuromuscular junction, resulting in muscle weakness and threat to life in severe cases. Over 85% of patients will progress to generalized myasthenia gravis within 24 months after disease onset and experience extreme fatigue and significant difficulties in facial expressions, speech, swallowing and physical movement.
Currently, there is no definitive treatment available for MG. However, targeted biological therapies have emerged as an area of special interest by virtue of their effectiveness, precision and safety.
Novel Dual-Target Mechanism
Telitacicept is a BLyS/APRIL dual-target fusion protein independently developed by RemeGen. As a novel dual-target fusion protein directed at B cells, Telitacicept simultaneously targets B-lymphocyte stimulator (BLyS) and a proliferation-inducing ligand (APRIL). This mechanism can effectively inhibit the excessive activation of B cells and plasma cells, thereby reducing the production of pathogenic antibodies associated with MG and offering significant therapeutic benefits.
Research has shown that B cell targeted therapies can significantly lower the relapse rate and the drug discontinuation rate among MG patients, compared to conventional immunotherapy regimens.
Expanding Global Development
Beyond the Chinese regulatory milestone, RemeGen announced on August 5, 2024, that it had achieved the milestone of First Patient In in the US for its global multi-center Phase III clinical trial on MG. The drug has received breakthrough therapy designation from China's NMPA, as well as orphan drug designation and fast track designation from the US FDA.
Telitacicept has been approved for two indications in China, namely systemic lupus erythematosus (SLE) and rheumatoid arthritis (RA), demonstrating the versatility of its dual-target mechanism across autoimmune conditions.
