Roche announced that the European Medicines Agency (EMA) has validated the company’s Marketing Authorisation Application (MAA) for satralizumab for the treatment of adult and adolescent patients with neuromyelitis optica spectrum disorder (NMOSD), granting it Accelerated Assessment. Validation confirms that the submission is complete and signifies the MAA is under review by the EMA’s Committee for Medicinal Products for Human Use (CHMP). The U.S. Food and Drug Administration (FDA) has also accepted the company’s Biologics License Application (BLA) for satralizumab. The CHMP recommendation and the FDA decision are expected in 2020.
“People living with NMOSD experience unpredictable relapses that can cause permanent neurological damage, and although there have been significant strides recently in understanding the disease, more approved options are needed with different treatment approaches. Satralizumab has shown robust efficacy sustained for 96 weeks and significantly reduced the risk of relapse across a broad patient population, while offering self-administered subcutaneous dosing every four weeks,” said Levi Garraway, M.D., Ph.D., Roche’s Chief Medical Officer and Head of Global Product Development. “The FDA and EMA’s acceptances of the satralizumab applications bring us one step closer to providing a new medicine to thousands of people impacted by NMOSD, and we are working with the health authorities to make satralizumab available as soon as possible.”
Accelerated Assessment reduces the timeframe for the EMA and CHMP to review the marketing authorisation, signifying the treatment is of major interest for public health or therapeutic innovation. The FDA previously granted Breakthrough Therapy Designation to satralizumab for the treatment of NMOSD in December 2018. In addition, satralizumab has been granted Priority Review in Canada and Switzerland, and designated as an orphan drug in the U.S., Europe and Japan.
If the EMA’s CHMP adopts a positive opinion that is endorsed by the European Commission, satralizumab will be granted a marketing authorisation valid in all 28 member states of the EU.
The satralizumab applications are based on positive results from two phase III studies, SAkuraStar and SAkuraSky, evaluating the efficacy and safety of satralizumab as a monotherapy and in combination with baseline immunosuppressant therapy, respectively. In the SAkuraStar study, satralizumab monotherapy achieved a 55% reduction in the risk of relapses compared to placebo in the overall study population of aquaporin-4 antibody (AQP4-IgG) seropositive and seronegative patients (Hazard Ratio [HR]=0.45, 95% Confidence Interval [CI]: 0.23-0.89; p=0.0184). Satralizumab achieved a 74% reduction (HR=0.26, 95% CI: 0.11-0.63; p=0.0014) in the larger (~67%) subgroup of AQP4-IgG seropositive patients, who tend to experience a more severe disease course. In the overall satralizumab-treated population, 76.1% were relapse-free at 48 weeks, and 72.1% relapse-free at 96 weeks, compared to 61.9% and 51.2% with placebo, respectively. Data from the AQP4-IgG seropositive subgroup showed that 82.9% were relapse-free at 48 weeks and 76.5% relapse-free at 96 weeks when treated with satralizumab, compared to 55.4% and 41.1% with placebo, respectively.
Additionally, the SAkuraSky study data showed a 62% reduction in the risk of relapses compared to placebo in the overall study population (HR=0.38, 95% CI: 0.16-0.88; p=0.0184) when used in combination with baseline immunosuppressant therapy, and a 79% reduction in the risk of relapses in AQP4-IgG seropositive patients (HR=0.21, 95% CI: 0.06-0.75; p=0.0086). In the overall satralizumab-treated population, 88.9% were relapse-free at 48 weeks, and 77.6% were relapse-free at 96 weeks, compared to 66.0% and 58.7% with placebo, respectively. Data from the AQP4-IgG seropositive subgroup showed that 91.5% were relapse-free at 48 and 96 weeks when treated with satralizumab, compared to 59.9% and 53.3% with placebo, respectively.
Overall, the proportion of patients with serious adverse events was similar between the satralizumab and placebo treatment groups in both studies. A lower rate of infections (including serious infections) was observed in patients treated with satralizumab compared with the placebo group. Most adverse events were mild to moderate, and the most common adverse events in the satralizumab group were urinary tract infection and upper respiratory tract infection in the SAkuraStar study and upper respiratory tract infection, nasopharyngitis (common cold) and headache in the SAkuraSky study.