FDA approves Enspryng to treat neuromyelitis optica spectrum disorder.- Genentech/Roche

Genentech, a member of the Roche Group announced that the FDA has approved Enspryng (satralizumab-mwge) as the first and only subcutaneous treatment for adults living with anti-aquaporin-4 (AQP4) antibody positive neuromyelitis optica spectrum disorder (NMOSD). NMOSD is a rare, lifelong and debilitating autoimmune disorder of the central nervous system, often misdiagnosed as multiple sclerosis, that primarily damages the optic nerve(s) and spinal cord, causing blindness, muscle weakness and paralysis. NMOSD is commonly associated with pathogenic antibodies (AQP4-IgG) that target and damage a specific cell type, called astrocytes, resulting in inflammatory lesions of the optic nerve(s), spinal cord and brain. AQP4-IgG antibodies are detectable in the blood serum of around 70-80% of NMOSD patients, and these patients tend to experience a more severe disease course. Although most cases of NMOSD can be confirmed through a diagnostic test, up to 30% of people living with the condition are still frequently misdiagnosed with multiple sclerosis. Enspryng is a humanized monoclonal antibody and the only approved therapy for NMOSD designed to target and inhibit interleukin-6 (IL-6) receptor activity, believed to play a key role in the inflammation associated with NMOSD. The treatment was designed using novel recycling antibody technology, which, compared to conventional technology, allows for longer duration of antibody circulation and subcutaneous dosing every four weeks. Enspryng can be administered in the home by a person living with NMOSD or a caregiver following training from a healthcare provider. Enspryng treatment is administered every four weeks after an initial loading dose. Enspryng will be available in the United States in two weeks. Genentech is committed to helping patients access the medicines prescribed by their physician. For people with NMOSD, the Enspryng Access Solutions team is available to answer questions, provide product education, injection training and help families understand insurance coverage and navigate appropriate financial assistance options to start and stay on Enspryng. Patients can call 1-844-NSPRYNG (844-677-7964) to speak to a Patient Navigator or visit http://www.Enspryng.com. FDA approval is based on results from one of the largest pivotal clinical trial programs undertaken for this rare neurological disorder.This approval is supported by results from two randomized controlled Phase III clinical trials, the SAkuraStar and SAkuraSky studies, in which Enspryng demonstrated robust and sustained efficacy and a favorable safety profile in adults with AQP4 antibody positive NMOSD. Results were sustained for 96 weeks, significantly reducing the risk of relapse compared with placebo as a monotherapy and when used concurrently with baseline immunosuppressant therapy (IST), which has commonly been used to manage NMOSD symptoms associated with relapses. In the SAkuraStar monotherapy study’s AQP4 antibody positive subgroup , 76.5% of Enspryng-treated patients were relapse-free at 96 weeks, compared to 41.1% with placebo. In the SAkuraSky study, which evaluated Enspryng when used concurrently with baseline IST , 91.1% of Enspryng-treated AQP4 antibody positive subgroup patients were relapse-free at 96 weeks, compared to 56.8% with placebo. The primary endpoint of both SAkuraStar and SAkuraSky was time to first protocol-defined relapse (PDR) adjudicated by an independent review committee in the double-blind period.