Safety, efficacy, and tolerability of efgartigimod in patients with generalised myasthenia gravis (ADAPT): a multicentre, randomised, placebo-controlled, phase 3 trial

SPS commentary:

The primary endpoint of study was proportion of acetylcholine receptor antibody-positive patients who were Myasthenia Gravis Activities of Daily Living (MG-ADL) responders (≥2-point MG-ADL improvement sustained for ≥4 weeks) in the first treatment cycle. The researchers note that individualised dosing based on clinical response was a unique feature of the study and translation to clinical practice with longer term safety and efficacy data will be further informed by the ongoing open-label extension.

According to a commentary, three promising biological drugs are now used for or expected to be safe and efficacious in the management of patients with refractory myasthenia gravis: rituximab, eculizumab, and efgartigimod. It notes that one question raised by these results is whether efgartigimod is more effective than eculizumab in patients with acetylcholine receptor-positive myasthenia gravis. It compares and contrasts the ADAPT trial (efgartigimod) with the phase 3 REGAIN trial (eculizumab) and adds that further study is necessary to compare efficacy between rituximab and efgartigimod for patients with MUSK-positive myasthenia gravis. It suggests biological drugs should be preferentially used as the treatment for patients with refractory myasthenia gravis, although the definition of refractory myasthenia gravis is different depending on the criteria used.