Cemdisiran Safe to Take, Has Low Treatment Burden in gMG: Tuan Vu, MD

Tuan Vu, MD, professor and division director of Neuromuscular Medicine & Electromyography at USF Health, discussed the safety of cemdisiran (Regeneron Pharmaceuticals) and its lighter treatment burden on patients living with generalized myasthenia gravis (gMG).

New results from the NIMBLE phase 3 randomized, double-blinded, controlled trial (NCT05070858) have found that cemdisiran was more effective in treating gMG compared with placebo, pozelimab monotherapy, and the combination of cemdisiran and pozelimab (Veopoz; Regeneron Pharmaceuticals). Patients had improved scores in both the Quantitative Myasthenia Gravis score and the Myasthenia Gravis Activities of Daily Living scale through 24 weeks. The combination of cemdisiran and pozelimab also met these primary and secondary end points, but the monotherapy of cemdisiran was found to be more effective overall.

In an interview with The American Journal of Managed Care^®, Vu explained that cemdisiran worked quickly, showing separation in efficacy as early as 2 weeks after initial treatment. He acknowledged that this brand of C5 inhibitors can lower the ability to fight bacteria.

“We did not see any case of that during this trial,” he said.

Vu also described how these results provide patients with new options for treatment. The subcutaneous delivery of cemdisiran allows patients to receive their treatment in just a few short minutes, and the gaps in treatment allow patients to continue to live their lives outside of treatment.

“In between treatments, the patient is free to do whatever: work, travel, and so on,” he said.

Vu emphasized that the adverse effects of the treatment were very tolerable, and the treatment was safe to take, making cemdisiran an appealing alternative for those living with gMG.

These results were presented as part of the 2026 American Academy of Neurology Annual Meeting, held in Chicago, Illinois, between April 18 and April 22, 2026. The results were presented as part of the presentation of new results in the neurology space throughout the weekend.