The findings of new research raises serious questions about the effectiveness of Soliris, suggesting that the immunosuppressant may be no better at fighting off debilitating muscle weakness than a placebo.
In a study published in the December issue of the medical journal The Lancet Neurology, researchers from the University of North Carolina at Chapel Hill evaluated the use of Soliris for treatment of a degenerative muscle disease known as myasthenia gravis, indicating that any benefits were statistically insignificant when compared to no treatment at all.
The findings came from a phase 3 randomized, double-blinded clinical trial involving 76 hospitals in 17 countries. During the study, 62 patients were treated with Soliris, and 63 with a placebo. Researcher conducted the study for 26 weeks to look for changes in a test meant to measure the progression of the disease, known as the Myasthenia Gravis-Activities of Daily Living (MG-ADL) score.
According to the researchers, score changes between patients given Soliris and the placebo patients were statistically insignificant. Side effects in both groups were similar, though Soliris patients had less reports of worsening conditions and less need for rescue therapy than the placebo group.
Soliris (eculizumab) is an immunosuppressant used to treat atypical hemolytic uremic syndrome and paroxysmal noctornal hemoglobinuria.
Side effects of Soliris have been linked to a 1,000-fold to 2,000-fold increased risk of meningococcal disease, resulting in the implementation of an FDA-required Risk Evaluation and Mitigation Strategy (REMS), which is designed to educate doctors and patients about the potential Soliris side effects and to detect early signs of infection. That strategy also includes steps that ensure patients receive the meningococcal vaccine before beginning Soliris treatment.
It already carries a black box warning about the risk of meningococcal infection, which is the strongest warning the FDA can review. However, that same black box warning advises health care professionals to “immunize patients with meningococcal vaccines at least 2 weeks prior to administering the first dose of Soliris, unless the risks of delaying Soliris therapy outweigh the risk of developing a meningococcal infection.”
Now, the U.S. Centers for Disease Control and Prevention (CDC), warns that even vaccination may not be enough to prevent meningococcal infections due to Soliris side effects.
The CDC and many doctors and health agencies around the world are now recommending that patients be placed on antimicrobial prophylaxis for as long as they take Soliris. For many patients, that means taking antimicrobial treatments for the rest of their lives.
Given the potential Soliris risks, the lack of evidence that the drug provides benefits for treatment of myasthenia gravis raise concerns about the potential use for this indication.