Zilucoplan for treating generalised myasthenia gravis


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Interventions: Zilucoplan (RA101495)
Indications: Myasthenia gravis (MG)
Therapeutic Areas: Musculoskeletal System
Year: 2021

Zilucoplan is currently in clinical development for adult patients with class II-IV generalized Myasthenia Gravis (gMG) who are Acetylcholine Receptor (AChR) antibody positive. Myasthenia gravis (MG) is a rare and chronic autoimmune disease. MG results from an abnormal immune reaction in which the body’s natural immune defences (i.e., antibodies) inappropriately attack certain receptors in muscles, causing debilitating and potentially life-threatening muscle weakness. Currently there are no medicinal products recommended by NICE specifically for the treatment of gMG, and medicines that are used can take a long time to work and result in side-affects.
Zilucoplan is a synthetic peptide that works by attaching to and blocking the C5 complement protein, one of the proteins of the 'complement system', which is part of the body's defence system. By blocking the C5 complement protein, zilucoplan is expected to prevent the complement system from damaging the blood cells, thereby helping to relieve the symptoms of the disease. Zilucoplan is administered via subcutaneous (SC) injection. If licenced, zilucoplan will provide a treatment option for those with gMG who are AChR antibody positive, reducing the treatment and disease burden in these patients.