EMA has recommended granting a marketing authorisation in the European Union (EU) for Enspryng (satralizumab) for the treatment of neuromyelitis optica spectrum disorders (NMOSD) in adults and adolescents from 12 years of age who are positive for anti-aquaporin-4 antibodies (AQP4-IgG).
NMOSD is a rare and life-threatening condition and most commonly affects the optic nerves and spinal cord. This disorder can lead to reduction or loss of vision, loss of sensation, loss of bowel and bladder control, weakness and paralysis of the arms and legs. NMOSD is thought to be caused by an abnormal reaction of the immune system that causes damage to healthy nerve cells. It is characterised by relapsing attacks, with symptoms coming back periodically. It is estimated that NMOSD affects approximately 1-2 in 100,000 people in the EU.
Enspryng works by reducing and preventing the attacks caused by NMOSD. Satralizumab, the active substance contained in Enspryng, is an antibody designed to block the inflammatory effects of interleukin-6 receptor (IL-6), which is involved in the pathogenesis of the NMOSD.
Enspryng will be available as a pre-filled syringe and will be administered as a solution through an injection under the patient’s skin (subcutaneously). The first three injections are given two weeks apart followed by one injection every four weeks. It can be used on its own or in combination with medicines that reduce the activity of the immune system (immunosuppressive therapy).
The opinion of EMA’s human medicines committee (CHMP) is mainly based on two randomised clinical studies which involved a total of 184 patients. The clinical studies showed that the chance of a relapse happening in 119 patients who were AQP4+ and received Enspryng alone or in combination with immunosuppressive therapy was a quarter of that in the control group receiving placebo alone or in combination with other immunosuppressive therapy.
The most common side effects observed in clinical trials were headache, joint pain, white blood cells count decreased and reactions at the site of injection.
The opinion adopted by the CHMP is an intermediary step on Enspryng’s path to patient access. The opinion will now be sent to the European Commission for the adoption of a decision on an EU-wide marketing authorisation. Once a marketing authorisation has been granted, decisions about price and reimbursement will take place at the level of each Member State, taking into account the potential role or use of this medicine in the context of the national health system of that country.