F. Hoffmann-La Roche Ltd: CHMP recommends EU approval of Roche's ENSPRYNG (satralizumab) for adults and adolescents with neuromyelitis optica spectrum disorder (NMOSD)

• If approved, ENSPRYNG will be the first and only treatment available to both adults and adolescents from 12 years of age with anti-aquaporin-4 antibody (AQP4-IgG) seropositive NMOSD in the EU
• ENSPRYNG is the only subcutaneous treatment option for NMOSD that can be administered at home every four weeks
• Recommendation is based on results from the two pivotal Phase III SAkuraStar and SAkuraSky studies, in which ENSPRYNG demonstrated robust and sustained efficacy in reducing the risk of relapse and a favourable safety profile
• NMOSD is a rare, lifelong and debilitating autoimmune disorder of the central nervous system that can cause blindness, muscle weakness and paralysis
  

Basel, 23 April 2021 - Roche (SIX: RO, ROG; OTCQX: RHHBY) announced today that the European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) has recommended the approval of ENSPRYNG (satralizumab) as the first subcutaneous treatment option for adults and adolescents from 12 years of age living with anti-aquaporin-4 antibody (AQP4-IgG) seropositive neuromyelitis optica spectrum disorder (NMOSD), as a monotherapy or in combination with immunosuppressive therapy (IST). AQP4-IgG are present in around 70-80% of people with NMOSD, who tend to experience a more severe disease course.

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 permanent blindness, muscle weakness and paralysis. The disease is characterised by unpredictable relapses and severe disability often occurs following the first NMOSD attack, accumulating with each subsequent relapse. Preventing these relapses is the primary goal for disease management. ENSPRYNG has been recommended for use in people who have only experienced a single NMOSD attack and adolescents, currently unserved NMOSD populations, as well as those with more advanced disease.

"Today's positive CHMP opinion is an important step toward bringing ENSPRYNG to people in the EU living with NMOSD who have limited treatment options," said Levi Garraway, M.D., Ph.D., Chief Medical Officer and Head of Global Product Development. "ENSPRYNG has been shown to reduce the risk of relapse significantly, while also offering a favourable safety profile. Additionally, if approved, ENSPRYNG would be the first and only treatment that can be self-administered subcutaneously at home following appropriate training."

ENSPRYNG is a humanised monoclonal antibody 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 by Chugai, a member of the Roche Group, using novel recycling antibody technology which, compared to conventional technology, allows for longer duration of antibody circulation and subcutaneous dosing every four weeks after an initial loading dose.

The CHMP recommendation is based on the results of the Phase III SAkuraStar and SAkuraSky studies in which ENSPRYNG showed robust and sustained efficacy results in reducing the risk of relapse and a favourable safety profile in people with AQP4-IgG seropositive NMOSD. A final decision regarding the approval is expected from the European Commission in the near future.

About SAkuraStar and SAkuraSky in NMOSD
SAkuraStar was a pivotal Phase III study evaluating the efficacy and safety of ENSPRYNG monotherapy administered to adults with neuromyelitis optica spectrum disorder (NMOSD). In the anti-aquaporin-4 antibody (AQP4-IgG) seropositive subgroup, 83% treated with ENSPRYNG remained relapse free at 48 weeks, compared with 55% of those treated with placebo. At 96 weeks, 77% of those treated with ENSPRYNG remained relapse free, compared with 41% with placebo.

SAkuraSky was a pivotal Phase III study evaluating the efficacy and safety of ENSPRYNG in combination with baseline immunosuppressive therapy in adults and adolescents with NMOSD. Overall, 92% of AQP4-seropositive participants receiving ENSPRYNG in combination with immunosuppressants remained relapse free at 48 and 96 weeks, compared with 60% and 53% with placebo at 48 and 96 weeks.

The primary endpoint of both the SAkuraStar and SAkuraSky studies was time to first protocol-defined relapse (PDR) adjudicated by an independent review committee in the double-blind period.

ENSPRYNG demonstrated a favourable safety and tolerability profile in the Phase III studies.

The most common adverse reactions observed in the safety population were: headache, arthralgia, white blood cell count decrease, hyperlipidaemia and injection-related reactions.

About neuromyelitisoptica spectrum disorder.

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