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News
A new siRNA therapy shows promise for myasthenia gravis. Cemdisiran, which targets complement component 5, dramatically reduced hospitalizations (3.8% vs. 15.3%) and myasthenic crises compared to placebo in the phase 3 NIMBLE trial. Both the FDA and EMA have accepted regulatory applications, with an FDA decision expected in November 2026.
Cemdisiran, an investigational small interfering RNA (siRNA) therapy that reduces complement component 5 (C5) production in the liver, significantly cut hospitalizations and myasthenic crises compared to placebo in a prespecified exploratory analysis of the phase 3 NIMBLE trial. Presented at the 2026 American Association of Neuromuscular and Electrodiagnostic Medicine meeting, the findings add to growing evidence that cemdisiran offers meaningful clinical benefits for patients with generalized myasthenia gravis (MG).
The drug was generally well-tolerated, with upper respiratory tract infections being the most common adverse event. Notably, the results suggest clinical benefits may be achievable without complete complement blockade — potentially preserving some immune defense against encapsulated bacteria, a possible advantage over existing C5 inhibitors.
By the Numbers:
Why it matters: With FDA and EMA regulatory applications already accepted — and an FDA decision expected in November 2026 — cemdisiran could soon offer a new subcutaneous treatment option for the roughly 95% of generalized MG patients who carry anti-acetylcholine receptor antibodies, a population with significant unmet need.
