rozanolixizumab for treating antibody-positive generalised myasthenia gravis

Rozanolixizumab for treating antibody-positive generalised myasthenia gravis

Myasthenia gravis is a rare chronic autoimmune disorder affecting the post-synaptic neuromuscular junction, primarily mediated by pathogenic immunoglobulin G (IgG) targeting specific proteins like acetylcholine receptor (AChR), muscle-specific tyrosine kinase (MuSK), and low-density lipoprotein receptor-related protein 4 (LRP4)

Rozanolixizumab, is a human IgG4 neonatal Fc receptor (FcRn) inhibitor, that enhances the degradation of pathogenic IgG (1):

• facilitates the degradation of IgG via the lysosomal pathway while simultaneously inhibiting the recycling of IgG back into circulation, thereby reducing overall IgG concentrations

NICE state that (2):

• rozanolixizumab, as an add-on to standard treatment, can be used as an option to treat generalised myasthenia gravis in adults who test positive for anti-acetylcholine receptor (AChR) or anti-muscle-specific tyrosine kinase (MuSK) antibodies. It can only be used if:
  • the condition is classified as Myasthenia Gravis Foundation of America (MGFA) class 2 to 4a
  • the condition is uncontrolled after 2 or more treatments, excluding acetylcholinesterase inhibitors, and
  • intravenous immunoglobulin (IVIg) or plasma exchange (PLEX) would otherwise be offered, or has been tried and stopped because of side effects or because it did not work well enough

The NICE committee noted that “..Clinical trial evidence suggests that rozanolixizumab plus standard treatment reduces symptoms and improves people's ability to carry out their normal activities compared with standard treatment alone. But it is uncertain if this improvement lasts in the longer term. Indirect comparisons suggest that rozanolixizumab works better than IVIg or PLEX, but the extent of the benefit is uncertain…”

Reference:

1. Matic A, Bril V. Rozanolixizumab for Myasthenia Gravis: a breakthrough treatment and future prospects. Immunotherapy. 2025 Apr;17(5):309-316.
2. NICE (May 2026). Rozanolixizumab for treating antibody-positive generalised myasthenia gravis.