Rare Daily Staff

The European Commission granted Roche Marketing Authorization for Enspryng as the first subcutaneous treatment option for adults and adolescents from 12 years of age with the rare disease neuromyelitis optica spectrum disorder.

Enspryng is approved for patients with anti-aquaporin-4 antibody (AQP4-IgG) seropositive NMOSD as a monotherapy or in combination with immunosuppressive therapy. It is the first and only NMOSD treatment that is administered subcutaneously every four weeks, allowing home-dosing after appropriate training.

NMOSD is a rare, lifelong, and debilitating autoimmune condition of the central nervous system that primarily damages the optic nerve(s) and spinal cord, causing permanent blindness, muscle weakness and paralysis. People with NMOSD experience unpredictable, severe relapses directly causing cumulative, permanent, neurological damage and disability. In some cases, relapse can result in death. NMOSD affects more than 10,000 people in Europe, up to 15,000 people in the United States, and approximately 200,000 people worldwide. NMOSD can affect individuals of any age, race and gender, but is most common among women in their 30s and 40s and appears to occur at higher rates in people of African or Asian background.

“With the approval of ENSPRYNG, we now have a treatment option with a favourable safety profile that significantly reduces relapses in AQP4-IgG seropositive adults and adolescents after their first NMOSD attack or in more advanced disease, either as a monotherapy or in combination with IST. Importantly, people with NMOSD now have the flexibility to administer treatment at home, which may alleviate the need to travel for hospital appointments,” said Friedemann Paul, professor of clinical neuroimmunology, Charité Universitätsmedizin Berlin.

Enspsryng is a humanized monoclonal antibody designed to bind to and block the interleukin-6 (IL-6) receptor, a central driver of the inflammation associated with NMOSD. The treatment was designed by Chugai, a member of the Roche Group, using novel recycling antibody technology. When compared to conventional antibodies, recycling antibody technology enables the medicine to remain in the bloodstream for a longer period of time and bind repeatedly to its target–maximally sustaining IL-6 suppression in a chronic disease like NMOSD and enabling subcutaneous dosing every four weeks.

The EC approval is supported by results from two phase 3 studies, in which Enspryng showed robust and sustained efficacy in reducing the risk of relapse in people with AQP4-IgG seropositive NMOSD. AQP4-IgG are present in around 70 to 80 percent of people with NMOSD, who tend to experience a more severe disease course compared to those not expressing AQP4-IgG antibodies.

“Building on our growing scientific understanding of neuroimmunological conditions, we are confident Enspryng can transform how people with NMOSD are treated by fitting into their day-to-day lives,” said Levi Garraway, chief medical officer and head of global product development at Roche.

EU approval follows approval in the United States and Japan. Other drugs approved for NMOSD include Alexion’s Soliris and Horizon therapeutics’ Uplizna.

Photo: Levi Garraway, chief medical officer and head of global product development at Roche

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