FDA Approves UCB Generalized Myasthenia Gravis Treatment

FDA announced approval of Rystiggo (rozanolixizumab-noli) as a treatment for generalized myasthenia gravis (gMG) in adult patients who are anti-acetylcholine receptor (AchR) or anti-muscle-specific tyrosine kinase (MuSK) antibody positive on June 27, 2023. Rozanolixizumab-noli is a subcutaneous administered, humanized monoclonal antibody injection designed to accelerate the catabolism of antibodies and reduce the concentration of pathogenic immunoglobulin G (IgG) autoantibodies. The approval was granted to Union Chimique Belge (UCB), a Belgian biopharmaceuticals company.

According to a UCB press release, gMG is a rare, chronic, autoimmune disease characterized by dysfunction and damage at the neuromuscular junction (NMJ). Pathogenic IgG autoantibodies can impair synaptic transmission at the NMJ by targeting specific proteins on the post-synaptic membrane, disrupting the ability of nerves to stimulate the muscle; this ultimately results in weaker muscle contractions.

FDA approval was based on data from the Phase III MycarinG study, whose primary efficacy endpoint was comparison of the change from baseline between treatment groups in the MG-ADL total score, a measurement tool which assesses the impact of gMG on daily functions, at Day 43. A statistically significant difference favoring rozanolixizumab-noli over placebo (-3.4 to -0.8) was observed in the MG-ADL total score change from baseline.

“No two people living with gMG experience the disease in the same way, so we can’t take a ‘one size fits all’ approach to disease management,” said Iris Loew-Friedrich, executive vice-president and chief medical officer, UCB, in the press release. “Disease management should be based on the clinical needs and preferences of the individual patient, and the aim of treatment is to help restore that patient’s ability to carry out activities of daily living. The approval of rozanolixizumab-noli means doctors have an additional approved treatment option for their gMG patients who have not yet found a treatment that meets their needs.”

FDA reviewed rozanolixizumab-noli under the priority review indication. The treatment is also currently being evaluated by the European Medicines Agency and the Japanese Pharmaceuticals and Medical Devices Agency.

Source: UCB