Roche's IL-6 Receptor Inhibitor Enspryng Receives First Approval in Asia for Neuromyelitis Optica Spectrum Disorder

Today, Roche announced that the Japanese Ministry of Health, Labour and Welfare (MHLW) has approved the marketing of Enspryng (satralizumab), an IL-6 receptor inhibitor developed by the company, for the prevention of disease relapse in adult and pediatric patients with neuromyelitis optica spectrum disorder (NMOSD) who are positive for anti-aquaporin-4 (AQP4) antibodies. This marks the first approval of this innovative therapy in Asia. Roche has submitted marketing applications for this new drug to the United States, the European Union, and China. In China, the application has been included in the list of proposed priority reviews, which is expected to accelerate the approval and launch of this innovative therapy in the country.

Neuromyelitis optica spectrum disorder (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 of the optic nerve 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 neurological damage and disability. Patients suffer from 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 central nervous system (CNS), approximately one-third of patients do not have these autoantibodies. 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. Enspryng, developed by Roche, is a fully humanized monoclonal antibody against the IL-6 receptor. It inhibits the IL-6 signaling pathway, thereby suppressing inflammation and the production of AQP4-targeting autoantibodies. Administered via subcutaneous injection every four weeks, Enspryng offers a convenient treatment option for patients and caregivers. The U.S. FDA has granted this therapy Breakthrough Therapy Designation.

The approval of Enspryng in Japan was based on the results of two pivotal Phase 3 clinical studies. These studies evaluated the efficacy and safety of Enspryng as monotherapy and as an add-on therapy to standard of care, respectively. The trial results demonstrated:

The SakuraStar study results showed that, compared with the placebo group, patients receiving Enspryng monotherapy had a 55% reduction in the risk of relapse. Furthermore, in the subgroup of patients positive for AQP4 autoantibodies, those receiving Enspryng monotherapy experienced a 74% reduction in relapse risk.

Research results from SAkuraSky showed that, compared with the placebo group, patients receiving Enspryng in combination with standard therapy had a 62% reduction in relapse risk. In the subgroup of patients positive for AQP4 autoantibodies, those receiving Enspryng in combination with standard therapy had a 79% reduction in relapse risk.

“Today’s approval marks the first approval of Enspryng in Asia, providing a new treatment option to reduce relapses in patients with NMOSD. These relapses can ultimately lead to irreversible disability, including blindness and paralysis,” said Dr. Levi Garraway, Chief Medical Officer and Head of Global Product Development at Roche. “Enspryng is the first and only approved therapy targeting the IL-6 receptor.”

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