Alexion Receives Positive CHMP Opinion for SOLIRIS

Alexion Pharmaceuticals announced the European Medicines Agency (EMA) Committee for Medicinal Products for Human Use (CHMP) has issued a positive opinion to extend the current marketing authorization of SOLIRIS® (eculizumab) to include the treatment of neuromyelitis optica spectrum disorder (NMOSD) in adult patients who are anti-aquaporin-4 (AQP4) antibody positive with a relapsing course of the disease. The European Commission will review the CHMP opinion and the final decision is anticipated within two months.

NMOSD is a rare, devastating, complement-mediated disorder of the central nervous system characterized by relapses, also referred to as attacks. Each attack results in stepwise accumulation of disability, including blindness and paralysis and sometimes premature death. NMOSD disproportionately strikes young women in the prime of their lives, with the average age of first onset at just 39 years. Previously known as Devic’s Disease, NMOSD is often confused with other neurological illnesses such as multiple sclerosis (MS), which can lead to delays in diagnosis and treatment with medicines that can worsen disease progression.

"Each and every NMOSD attack has the potential to result in serious, irreversible consequences like blindness or losing the ability to walk," said John Orloff, M.D., Executive Vice President and Head of Research and Development at Alexion. "This is why attack, or relapse, prevention is the primary treatment goal for people living with NMOSD. There are currently no treatments approved in Europe for NMOSD; this positive opinion brings us one step closer to being able to offer SOLIRIS as the first-approved therapy for this very severe condition."

The positive opinion is based on comprehensive results from the Phase 3 randomized, double-blind placebo controlled PREVENT trial, which were published in The New England Journal of Medicine and a long-term extension study (ECU-NMO-302), which is still underway. In the PREVENT study, patients with NMOSD who were anti-AQP4 antibody positive were treated with SOLIRIS (n=96) or placebo (n=47). The study met its primary endpoint of prolonging the time to first adjudicated relapse and reducing the risk of relapse. At 48 weeks, 98 percent of patients treated with SOLIRIS were relapse free compared to 63 percent of patients receiving placebo. Of the patients treated solely with SOLIRIS, without receiving other immunosuppressive therapies, 100 percent were relapse free at 48 weeks compared to 61 percent in the placebo group. Sustained effects were observed through 144 weeks of treatment. The safety profile of SOLIRIS was consistent with that seen for SOLIRIS in other clinical studies and real-world use in its three approved indications. The most common adverse events observed in the PREVENT study were upper respiratory tract infection (29 percent of patients in the SOLIRIS group vs. 13 percent in the placebo group), headache (23 vs. 23 percent), nasopharyngitis (21 vs. 19 percent) and nausea (17 vs. 26 percent). 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 U.S. Food and Drug Administration (FDA) approved SOLIRIS (eculizumab) for the treatment of Neuromyelitis Optica Spectrum Disorder (NMOSD) in adult patients who are anti-aquaporin-4 (AQP4) antibody positive on June 27, 2019. A supplemental New Drug Application currently is under review by regulatory authorities in Japan. SOLIRIS received Orphan Drug Designation (ODD) for the treatment of NMOSD in the U.S., EU and Japan.

NMOSD is a rare and severe, autoimmune, inflammatory disorder that attacks the central nervous system (CNS), in which complement activation due to anti-aquaporin-4 (AQP4) antibodies plays a significant role in the disease process. Patients with NMOSD experience unpredictable attacks, also referred to as relapses, which can cause irreversible damage to the optic nerve and spinal cord and can lead to long-term disability. The most common symptoms of NMOSD are optic neuritis and transverse myelitis. Optic neuritis can cause visual problems including blindness; Transverse myelitis can cause mobility problems including paralysis.

The disease primarily affects women, often in the prime of their lives, with an average age of onset of 39 years. The prevalence of NMOSD may be more common and more severe in non-Caucasian populations worldwide.

Complement activation by anti-AQP4 auto-antibodies can cause destruction of vital cells in the CNS, leading to demyelination and to the death of neurons, predominantly in the spinal cord and optic nerve. Approximately three quarters (73%) of all patients with NMOSD have AQP4 auto-antibodies. In patients with anti-AQP4 antibody positive NMOSD, the body’s own immune system can turn against itself to produce auto-antibodies against AQP4, a protein on certain cells in the eyes, brain and spinal cord that are critical for the survival of nerve cells. The binding of these anti-AQP4 auto-antibodies activates the complement cascade, another part of the immune system.

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