Anshiping®-the first and only drug with indications for the treatment of NMOSD landed for the first time in China, creating a new era of NMOSD treatment

Neuromyelitis optica spectrum disease (NMOSD) is a group of autoimmune-mediated central nervous system inflammatory demyelinating diseases mainly involving the optic nerve and spinal cord
.

Aquaporin 4-IgG (AQP4-IgG) is a NMOSD-specific disease marker.
About 70%-80% of NMOSD patients are AQP4-IgG positive.
AQP4-IgG positive indicates that the disease is prone to relapse
.

NMOSD patients accumulate disability due to frequent and severe disease recurrences.
Therefore, prevention of recurrence is the top priority of NMOSD treatment
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The disease was included in the first batch of 121 rare diseases in the country in May 2018
.

Previously, there were no drugs approved for NMOSD treatment indications in China, and the current status of treatment of NMOSD patients in China is not optimistic
.

On April 30, 2021, the National Medical Products Administration (NMPA) of China officially approved satrizizumab injection (trade name: Anshiping®) for the treatment of AQP4-IgG-positive NMOSD patients over 12 years old, becoming China The first approved NMOSD treatment indication drug in mainland China
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Thus, the treatment of NMOSD ushered in the era of standardized treatment
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Since its commercial launch at the end of November, patients in Guangdong and Sichuan have started to use satrizizumab injection
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So, in clinical practice, what benefits can satrizizumab bring to patients with NMOSD? 01 Excellent efficacy-combined with baseline immunotherapy, up to 91% of AQP4-IgG positive patients had no serious recurrence at 192 weeks.
Two multi-center phase III clinical studies for NMOSD patients were carried out globally by satrizizumab, respectively For SAkuraSky research and SAkuraStar research
.

In the double-blind (DB) phase of the above two studies, satrizizumab significantly reduced the risk of recurrence in patients with NMOSD
.

In the SakuraSky study, the 96-week relapse-free rate of satrizizumab combined with baseline immunosuppressive therapy in AQP4-IgG-positive patients was as high as 92%1
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In the SAkuraStar study, the recurrence-free rate of patients with AQP4-IgG positive satrizizumab was 77% after 96 weeks 2
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This year, the ECTRIMS conference published an extended phase study 3 to evaluate the long-term efficacy of satrizizumab in AQP4-IgG-positive patients from the SAkura study
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Efficacy measures include: relapse (iPDR) defined by the study protocol, severe iPDR, and continuous deterioration of the Extended Disability Status Scale (EDSS)
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The results of the study showed that during the open label expansion (OLE) phase of the SAkuraSky study and the SAkuraStar study, the efficacy of satrizizumab continued.
At the 192th week (3.
7 years), patients without iPDR, severe iPDR, or EDSS continued to worsen The proportion is very high (Figure 1, Figure 2)
.

Figure 1 SAkuraSky research results Figure 2 SAkuraStar research results 02 Safe and reliable-long-term safety, the risk of infection in the OST phase is similar to that in the DB phase, and there is no allergic reaction.
At this year's ECTRIMS conference, an extended phase study 4 was published.
Evaluated the long-term safety of satrizizumab in the intent-to-treat (ITT) population from the SAkura study
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The results of the study showed that the incidence of adverse events (AE) and severe AEs during the overall satrizizumab treatment (OST) phase was comparable to that of satrizizumab and placebo in the DB phase
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The overall infection rate and severe infection rate in the OST stage are similar to those in the DB stage
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No allergic reactions related to the study treatment were reported in either the DB phase or the OST phase of the SAkuraSky study and the SAkuraStar study
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In short, the long-term safety of satelizumab remained unchanged in the OST phase, and no new safety issues were observed in the OST and DB phases
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Table 1 SAkuraSky and SAkuraStar adverse events in the DB and OST stages (number of events/100 patient-years) 03 Easy to use-subcutaneous injection form, worry-free home use, high compliance 5 satrizizumab is given a loading dose for the first time (Subcutaneous injection at 0, 2, and 4 weeks), and repeat subcutaneous injection every 4 weeks thereafter
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With proper training, patients or caregivers can inject themselves at home, avoiding the worry of multiple visits to the hospital for injecting drugs
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Thereby improving the compliance and convenience of patient treatment
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04Experts interpret that satelizumab is the first drug approved for NMOSD treatment indication in mainland China, and the approval of this drug will undoubtedly become a milestone in NMOSD treatment
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Expert profile Professor Zhou Hongyu, chief physician, professor, and doctoral supervisor of the Department of Neurology, West China Hospital of Sichuan University.
Academic position: member of the Neuroimmune Disease Professional Committee of the Neurologist Branch of the Chinese Medical Doctor Association, standing member of the Immunology Branch of the Chinese Stroke Society, and member of the Neuroimmune Branch of the Chinese Society of Immunology The leader of the neuroimmunology group of the Neurology Professional Committee of Sichuan Medical Association entered the Department of Neurology of West China Hospital in 1995, and successively trained more than 30 postgraduates for doctoral and master's degrees; presided over 2 projects of the National Natural Science Foundation of China; 1 key research project of the Ministry of Science and Technology; The Provincial Department of Science and Technology supports 2 research projects, more than ten horizontal projects and many other scientific research projects
.

As the first author or corresponding author, he has published more than 70 papers.
Among them, more than 30 SCI papers have such a group of rare diseases.
They are most worried about the recurrence of the disease every year, because every time they relapse, they may lose their eyesight, mobility and even life
.

After 5 years of these patients, 50% of the patients need a wheelchair, 62% of the patients will be blind6, and 60.
4% of the patients cannot take care of themselves.
This is NMOSD
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The high recurrence and high disability of NMOSD seriously affect the treatment and prognosis of patients.
Therefore, preventing recurrence is the top priority of NMOSD treatment
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In addition to the difficulties in its own treatment, the disease also brings a great economic and psychological burden to patients and their families
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In recent years, some emerging therapeutic target monoclonal antibody drugs have continued to emerge, and RCT research results have shown significant efficacy, providing a higher evidence-based basis for the field of NMOSD treatment
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Three drugs have been officially approved by the US FDA or the European Union for the treatment of NMOSD, including: complement inhibitors, IL-6 receptor blockers, and B lymphocyte depleting agents
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On April 30, 2021, NMPA officially approved satrizizumab for the treatment of children over 12 years of age and adults with AQP4-IgG positive NMOSD patients, becoming the first approved NMOSD treatment indication drug in Mainland China
.

Two multi-center phase III clinical studies of satrizizumab for NMOSD patients carried out globally are the SAkuraSky study and the SAkuraStar study
.

These two studies confirmed that satrizizumab alone or combined with baseline immunosuppressive therapy can significantly reduce the risk of recurrence in patients with NMOSD
.

In addition, the safety of satrizizumab has also been confirmed by research, and the incidence of infection and severe infection is equivalent to that of the placebo group, and there are no serious allergic reactions and deaths
.

Two extended-phase studies published at this year's ECTRIMS conference also confirmed the long-term efficacy and long-term safety of satrizizumab in AQP4-IgG-positive patients from the SAkura study
.

Moreover, satrizizumab is a subcutaneous injection formulation, which is convenient to use and can be used at home, avoiding the trouble of visiting the hospital for multiple injections
.

As the first drug approved for NMOSD treatment indications in mainland China, satrizizumab fills the gap in the treatment of NMOSD in remission in China and will bring more benefits to NMOSD patients! References: 1.
Yamamura T, et al.
N Engl J Med.
2019 Nov 28;381(22):2114-2124.
2.
Traboulsee A, et al.
Lancet Neurol.
2020 May;19(5):402-412.
3.
Long-term efficacy of satralizumab in AQP4-IgG-seropositive NMOSD—Results from the open-label extension periods of SAkuraSky and SAkuraStar.
ECTRIMS 2021.
4.
Long-term safety of satralizumab in neuromyelitis optica spectrum disorder—Results from the open-label extension periods of SAkuraSky and SAkuraStar.
ECTRIMS 2021.
5.
Chinese instructions for satrizizumab 6.
Kessler RA, et al.
Neurol Neuroimmunol Neuroinflamm.
2016 Jul 28;3(5):e269.
7.
2019 Chinese Neuromyelitis Optician Spectrum Disease Patients Comprehensive Social Survey