We’re pleased to share the news that the European Medicines Agency (EMA) Committee for Medicinal Products for Human Use (CHMP) has issued a positive opinion regarding the use of eculizumab (Soliris) as a treatment for people with refractory generalised myasthenia gravis who are anti-acetylcholine receptor (AChR) antibody-positive. This particular type of myasthenia gravis (MG) is very rare and does not respond to conventional immunosuppressive therapy.
Eculizumab is an antibody that blocks part of the body’s immune response. It was tested in people with refractory generalised MG as part of a phase 3 study called REGAIN. Although some clinical improvements were reported, the study’s primary efficacy endpoint did not reach statistical significance. The primary endpoint is the main result that is measured at the end of a study to see if a drug or treatment was effective.
The pharmaceutical company, Alexion, submitted to the CHMP data from both the REGAIN study and a longer-term extension study, which remains ongoing.
Now that eculizumab has been given a positive opinion by the CHMP, the decision must be ratified by the European Commission. It is expected that this will be done in the third quarter of 2017. We will keep you updated of any developments.
Dr John Orloff, Executive Vice President and Head of Research & Development at Alexion, said in a press release:
Despite existing treatment options for gMG, patients with refractory gMG continue to suffer from severe symptoms and disease complications that significantly impact their daily lives. The positive CHMP opinion is a critical milestone in bringing Soliris to patients with refractory gMG who are anti-AChR antibody-positive and for whom physicians currently have no approved therapy.
Eculizumab already has approval in the EU for two ultra-rare conditions: paroxysmal nocturnal hemoglobinuria (PNH) and typical hemolytic uremic syndrome (aHUS).
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