An original mode of administration in the treatment of refractory myasthenia gravis

An international consortium of clinicians reports the results of a phase IIIAu cours d’un essai clinique de phase III, un médicament, pour lequel on a déterminé lors d’essais antérieurs l’innocuité et le dosage optimum (essais de phase I et II), est administré à un grand groupe de malades, sur une longue durée, dans le but d’évaluer son efficacité thérapeutique en la comparant à celle d’un traitement de référence ou un placebo. Il permet aussi de mettre en évidence les interactions indésirables et les effets secondaires du traitement à moyen terme. Au terme de cet essai, le médicament peut obtenir une autorisation de mise sur le marché. open-label trial designed to assess the benefit and tolerability of a monoclonal antibody, rozanolixizumab, administered by the patient either by manual pressure or via an infusion pump:

• 62 adults with generalised myasthenia gravis participated in the trial and were divided into two groups, with a crossover period.
• The trial lasted a total of 18 weeks, with the usual efficacy and safety criteria being used.
• 100% of patients were successful with this alternative method and 63% preferred to be able to control it themselves (by manual pressure).

As well tolerated as in a hospital setting and as effective in terms of clinical parameters, this alternative method of home administration could eventually become the norm for a larger number of patients.

Self-administration of rozanolixizumab via manual push and infusion pump methods in patients with generalised myasthenia gravis: a randomised, phase 3, open-label, crossover study. Bril V, Antozzi C, Berkowicz T et al. J Neurol. 2025 Oct