Roche's IL-6 Inhibitor Satralizumab Receives FDA and EMA Acceptance for NMOSD with Significant Reduction in Relapse Risk

Today, Genentech, a member of the Roche Group, announced that the U.S. Food and Drug Administration (FDA) has accepted its Biologics License Application (BLA) for satralizumab, an IL-6 antibody, for the treatment of adult and adolescent patients with neuromyelitis optica spectrum disorder (NMOSD). Meanwhile, the European Medicines Agency (EMA) has also accepted the Marketing Authorization Application (MAA) for satralizumab and granted it Accelerated Assessment status. The FDA and the EMA’s Committee for Medicinal Products for Human Use (CHMP) are expected to issue decisions on these applications in 2020.

NMOSD is a rare, disabling autoimmune disease of the central nervous system (CNS) that affects up to hundreds of thousands of people worldwide. The primary symptoms in patients are inflammatory lesions in the optic nerves and spinal cord. Patients with NMOSD often experience disease relapses, and repeated attacks by the immune system on their own tissues lead to progressive accumulation of nerve damage and disability. Patients may experience declines in vision, motor function, and quality of life, and severe NMOSD attacks can be fatal. Currently, there are no approved therapies for treating this disease.

Although NMOSD is considered a disease caused by autoimmune antibodies targeting AQP4 entering the CNS, approximately one-third of patients do not have autoimmune antibodies targeting AQP4. In recent years, the pro-inflammatory cytokine IL-6 has emerged as an important therapeutic target in the pathogenesis of NMOSD, triggering inflammatory cascades that lead to tissue damage and disability. Satralizumab, developed by Roche, is a fully humanized monoclonal antibody against IL-6. It inhibits the IL-6 signaling pathway, thereby suppressing inflammation and the production of autoimmune antibodies targeting AQP4. Administered via subcutaneous injection once every four weeks, satralizumab offers a convenient treatment option for patients and caregivers. Last December, the U.S. FDA granted Breakthrough Therapy Designation to this therapy.

▲Introduction to Satralizumab (Image source: Reference [2])

This application is based on the positive results from two pivotal Phase 3 clinical studies, named SakuraStar and SAkuraSky. These trials evaluated the efficacy and safety of satralizumab as monotherapy and in combination with standard therapy, respectively, in patients with neuromyelitis optica spectrum disorder (NMOSD). The results of the SakuraStar study showed that, compared with the placebo group, patients receiving satralizumab monotherapy had a 55% reduction in the risk of relapse. The relapse-free rates at 96 weeks and 48 weeks were 76.1% and 72.1%, respectively, whereas these rates in the placebo group were 61.9% and 51.2%. Furthermore, in the subgroup of patients positive for aquaporin-4 (AQP4) autoantibodies, satralizumab monotherapy reduced the risk of relapse by 74%. The relapse-free rates at 96 weeks and 48 weeks were 76.5% and 82.9%, respectively, compared with 41.1% and 55.4% in the placebo group.

Results from the SAkuraSky study showed that, compared with the placebo group, patients receiving satralizumab in combination with standard therapy had a 62% reduction in the risk of relapse. The relapse-free rates at 96 weeks and 48 weeks were 77.6% and 88.9%, respectively, whereas in the placebo group, these rates were 58.7% and 66%, respectively. In the subgroup of patients positive for AQP4 autoantibodies, those receiving satralizumab in combination with standard therapy had a 79% reduction in the risk of relapse. The relapse-free rates at 96 weeks and 48 weeks were 91.5%, whereas in the placebo group, these rates were 53.3% and 59.9%, respectively.

“Patients with NMOSD often experience disease relapses, which can lead to permanent neurological damage. Satralizumab has demonstrated sustained efficacy for up to 96 weeks in clinical trials and significantly reduced the risk of relapse across a broad patient population,” said Dr. Levi Garraway, Head of Global Product Development and Chief Medical Officer at Roche. “The acceptance of satralizumab’s marketing applications by the FDA and EMA marks an important step forward in our efforts to provide a new treatment option for patients with NMOSD. We look forward to working with regulatory authorities to make satralizumab available to NMOSD patients as soon as possible.”

References:

[1] FDA Accepts Genentech’s Biologics License Application for Satralizumab for Neuromyelitis Optica Spectrum Disorder, Retrieved October 30, 2019, from https://www.businesswire.com/news/home/20191029006190/en/

[2] Roche Pharma Day 2019. Retrieved September 16, 2019, from https://www.roche.com/dam/jcr:8f38992b-8977-46d3-8d92-f4f3f939245a/en/irp20190916.pdf

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