1 July 2019 - - The US Food and Drug Administration has approved Soliris (eculizumab) for the treatment of neuromyelitis optica spectrum disorder (NMOSD) in adult patients who are anti-aquaporin-4 antibody positive, US-based Alexion Pharmaceuticals, Inc. (NASDAQ: ALXN) said.
Approximately three quarters of all patients with NMOSD test positive for anti-AQP4 auto-antibodies.
The FDA approved Soliris following an expedited six-month priority review. NMOSD is a rare, severe autoimmune disease that attacks the central nervous system without warning.
These attacks, also referred to as relapses, can cause progressive and irreversible damage to the brain, optic nerve and spinal cord, which may lead to long-term disability.
Complement activation due to anti-AQP4 antibodies is one of the primary underlying causes of the destruction in these patients.
In the PREVENT trial, Soliris, a first-in-class complement inhibitor, demonstrated safety and efficacy and met its primary endpoint of prolonging the time to first adjudicated relapse and reducing the risk of relapse.
NMOSD disproportionately strikes young women in the prime of their lives, with the average age of first onset at just 39 years.
Race is also a significant risk factor for disability and mortality in NMOSD.
In the US, African Americans are over-represented among patients diagnosed with NMOSD and more likely to suffer more frequent and more severe attacks.
Previously known as Devic's Disease, NMOSD is often confused with other neurological illnesses such as multiple sclerosis, which can lead to delays in diagnosis and treatment with medicines that can worsen disease progression.
This approval is based on comprehensive results from the Phase 3 randomised, double-blind placebo controlled PREVENT trial, which were recently published in The New England Journal of Medicine.
In the study, patients with NMOSD who were anti-AQP4 antibody positive were treated with SOLIRIS or placebo.
At 48 weeks, 98 % of patients treated with SOLIRIS were relapse free compared to 63 % of patients receiving placebo.
This effect was observed through 144 weeks of treatment, with 96% of patients treated with SOLIRIS relapse free compared to 45 % of patients in the placebo arm. Soliris-treated patients experienced similar improvement in time to first adjudicated on-trial relapse with or without concomitant treatment.
Of the patients treated solely with Soliris, without receiving other immunosuppressive therapies,, 100% were relapse free at 144 weeks compared to 20% in the placebo group.
The safety profile of Soliris was consistent with that seen for Soliris in other clinical studies. The most common adverse events observed in the PREVENT study were upper respiratory tract infection (29% of patients in the Soliris group vs. 13% in the placebo group), nasopharyngitis (21 vs. 19%), diarrhea (16 vs. 15%), back pain (15 vs. 13%) and dizziness (15 vs. 13%).
The serious adverse events that were reported for more than one patient in either group were pneumonia (three patients in the Soliris group vs. one patient in the placebo group) and cellulitis, sepsis and urinary tract infection (two patients for each event in the Soliris group vs. no patient in the placebo group).
One patient receiving Soliris and concomitant supportive IST died from infectious pleural effusion.
The patient had an extensive history of pulmonary disease and was an active smoker.
No cases of meningococcal infection were observed in the study.
The European Medicines Agency and the Japanese Ministry of Health, Labour and Welfare are reviewing Alexion's applications to add the treatment of NMOSD to the marketing authorisations for Soliris in the European Union and Japan, respectively.
Soliris has received Orphan Drug Designation for the treatment of patients with NMOSD in the US, EU and Japan.
Soliris is a prescription medicine called a monoclonal antibody. Soliris is used to treat patients with a disease called Paroxysmal Nocturnal Hemoglobinuria. Soliris is used to treat adults and children with a disease called atypical Hemolytic Uremic Syndrome.