Ravulizumab Fast Track - access to treatments

Ravulizumab is a potential treatment for generalised myasthenia gravis developed by Alexion Pharmaceuticals.

Generalised myasthenia gravis is a long-term autoimmune disorder where the body’s own immune system mistakenly attacks healthy cells in the neuromuscular junction. This junction is a region where nerve cells transmit signals to muscle cells to result in muscle contraction. The main symptoms are weak muscles that tire easily, which makes regular activities such as chewing, swallowing, talking, and walking more challenging. 

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 generalised myasthenia gravis by causing over-activation of the complement cascade (a pathway in the immune system) that results in the immune system attacking cells in the neuromuscular junction. Blocking the C5 protein results in improved communication between the nerve cells and muscle cells in the NMJ, and therefore improving the symptoms of generalised myasthenia gravis.

The treatment is currently being reviewed by NICE. MDUK took part in a NICE scoping appraisal in April 2022, ahead of which we liaised with specialist neuromuscular clinicians. We are awaiting next steps and aim to work in partnership with people with myasthenia gravis to submit the patient voice as part of any future consultation/appraisal.