FDA Approves UCB Generalized Myasthenia Gravis Treatment
UCB’s rozanolixizumab-noli is approved for treatment of generalized myasthenia gravis in certain adult patient groups.
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
Trump Directs FDA, EPA to Reduce Regulatory Barriers to Domestic Pharma Manufacturing
May 7th 2025The White House is instructing FDA to increase fees for and inspections of foreign drug manufacturing plants and reduce the time required to approve such sites that will be newly constructed in the US.
New Cancer Research and Treatment Center in Development in London
May 1st 2025Aviva Capital Partners and developer Socius are investing £1 billion in a 12-acre site to be located at the London Cancer Hub next to The Institute of Cancer Research, London and The Royal Marsden NHS Foundation Trust’s Sutton site.
Regeneron Treatment for Multiple Myeloma Gets Conditional Marketing Approval from EC
April 29th 2025The indication is specific to patients who have received at least three prior therapies, including a proteasome inhibitor, immunomodulatory agent, and anti-CD38 monoclonal antibody, and have demonstrated disease progression on the last therapy.
