Ravulizumab for treating generalised myasthenia gravis

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

Ravulizumab is currently in clinical development for the treatment of adults with generalised myasthenia gravis (gMG). gMG is a long-term autoimmune disorder where the body’s own immune system mistakenly attacks healthy cells in the neuromuscular junction (NMJ). The NMJ is a region where nerve cells transmit signals to muscle cells to result in muscle contraction. The main symptom of gMG is weak muscles that tire easily, and this can negatively impact patient quality of life by making activities of daily living such as chewing, swallowing, talking and walking more challenging. 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.
Ravulizumab is a modified human antibody (a protein produced by the immune system) that is administered by intravenous (IV) infusion and works by blocking the protein C5. This protein is known to play a role in development of gMG by causing over-activation of the complement cascade (a pathway in the immune system) that results in the immune system attacking cells in the NMJ. Blocking the C5 protein results in improved communication between the nerve cells and muscle cells in the NMJ, and therefore improving the symptoms of gMG. If licensed, ravulizumab would be the first medicinal product approved by NICE for the treatment of gMG and reduce the treatment burden for these patients.