Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Satralizumab · 9 trials · 10 indications
Percentage of participants in the active TED population (i.e., participants who have active disease) who achieved a ≥ 2 mm reduction in proptosis from baseline (Day 1) to Week 24 in the study eye, with no corresponding deterioration of proptosis (≥ 2 mm increase) in the fellow eye have been reported. Percentages have been rounded off.
AE=any untoward medical occurrence in a participant administered a pharmaceutical product, regardless of causal relationship with it. AE can therefore be any unfavorable \& unintended sign, symptoms/disease temporally associated with use of a medicinal (investigational) product, whether or not considered related to it. SAE is any significant hazard, contraindication, or side effect that is fatal or life-threatening, requires hospitalization or prolongation of an existing hospitalization, results in persistent or significant disability/incapacity, is a congenital anomaly/birth defect, is medically significant or requires intervention. The first dosing visit in the current study or randomization visit in the parent studies (NCT02028884/NCT02073279) was considered as baseline for this outcome measure. All AEs from the time of randomization in the parent studies for satralizumab-treated participants are reported here.
An AESIs included potential drug induced liver injury and suspected transmission of an infectious agent by study drug defined as any organism, virus, or infectious particle (e.g., prion protein transmitting transmissible spongiform encephalopathy), pathogenic or non-pathogenic causing clinical symptoms or laboratory findings that indicate an infection in a participant exposed to a medicinal product. Selected AEs included infections that required treatments with IV antibiotics, antifungals, or antivirals; opportunistic infections that required treatment with oral antibiotics, antifungals, or antivirals and injection related reaction. The first dosing visit in the current study or randomization visit in the parent studies (NCT02028884/NCT02073279) was considered as baseline for this outcome measure. All AEs from the time of randomization in the parent studies for satralizumab-treated participants are reported here.
TFR was defined as time from randomization to first occurrence of relapse in the DB period. Protocol-defined relapse was occurrence of new or worsening neurological symptoms attributable to neurological neuromyelitis optica (NMO) or neuromyelitis optica spectrum disorder (NMOSD). Symptoms had to persist for \>24 hours and not be attributable to confounding clinical factors (e.g., fever, infection, injury, change in mood, adverse reactions to medications). New or worsening neurological symptoms that occurred \< 31 days following onset of a protocol-defined relapse were considered part of same relapse (i.e., if 2 relapses had onset days that were 30 days of one another, they were counted only as 1 relapse), and onset date used in analysis was the date of first relapse.
TFR was defined as time from randomization to first occurrence of relapse in the DB period. Protocol-defined relapse was occurrence of new or worsening neurological symptoms attributable to neurological neuromyelitis optica (NMO) or neuromyelitis optica spectrum disorder (NMOSD) as adjudicated by an independent clinical endpoint committee (CEC). Symptoms had to persist for \>24 hours and not be attributable to confounding clinical factors (e.g., fever, infection, injury, change in mood, adverse reactions to medications). New or worsening neurological symptoms that occurred \< 31 days following onset of a protocol-defined relapse were considered part of same relapse (i.e., if 2 relapses had onset days that were 30 days of one another, they were counted only as 1 relapse), and onset date used in analysis was the date of first relapse.
BMD of the LS is measured using DEXA.
| Arm | Type | Description |
|---|---|---|
| Cohort 1: Participants with body weight ≥10kg to <20kg | EXPERIMENTAL | Satralizumab will be administered SC Q6W in a cohort of at least 2 evaluable patients |
| Cohort 2 Participants with body weight ≥20kg to <40kg | EXPERIMENTAL | Satralizumab will be administered SC at Weeks 0, 2, 4, and Q4W thereafter. |
| Cohort 3 Participants with body weight ≥40kg | EXPERIMENTAL | Satralizumab will be administered SC at Weeks 0, 2, 4, and Q4W thereafter. |
| Satralizumab | EXPERIMENTAL | In the Part I period, participants will receive satralizumab every 4 weeks (q4w) followed by proptosis response-based individualized treatment in Part II of the study. |
| Placebo | PLACEBO_COMPARATOR | In the part I period, participants will receive placebo q4w followed by proptosis response-based individualized treatment in part II of the study. |
| NMDAR Autoimmune Encephalitis (AIE) Cohort | EXPERIMENTAL | Adults and adolescents with definite or probable NMDAR encephalitis will receive satralizumab as per the schedule specified in the protocol. As of Protocol Version 6, participants in Part 1 will transition to Part 2 at the time of primary analysis or at Week 52, whichever occurs first. |
| LGI1 AIE Cohort | EXPERIMENTAL | Adults with LGI1 encephalitis as per the schedule specified in the protocol. |
| NMDAR AIE Placebo Cohort | PLACEBO_COMPARATOR | Adults and adolescents with definite or probable NMDAR encephalitis will receive satralizumab placebo as per the schedule specified in the protocol. As of Protocol Version 6, participants in Part 1 will transition to Part 2 at the time of primary analysis or at Week 52, whichever occurs first. |
| LGI1 AIE Placebo Cohort | PLACEBO_COMPARATOR | Adults with LGI1 encephalitis will receive satralizumab placebo as per the schedule specified in the protocol. |
| Satralizumab Treatment | EXPERIMENTAL | Participants will receive satralizumab subcutaneously (SC) every 4 weeks (Q4W) |
| Satralizumab + Baseline Treatment | EXPERIMENTAL | Participants randomized to this arm for the double-blind period will receive satralizumab in addition to baseline treatment. The double-blind period ends when either the participant has a treated relapse or the total number of protocol-defined relapses confirmed by the Clinical Endpoint Committee (CEC) reaches 26. In the open-label extension period, the participant will receive (with or without baseline treatment) an SC injection of satralizumab at Weeks 0, 2, and 4, and Q4W thereafter, with the last study drug administration on or before 31 December 2021. |
| Placebo + Baseline Treatment | PLACEBO_COMPARATOR | Participants randomized to this arm for the double-blind period will receive placebo in addition to baseline treatment.The double-blind period ends when either the participant has a treated relapse or the total number of protocol-defined relapses confirmed by the Clinical Endpoint Committee (CEC) reaches 26. In the open-label extension period, the participant will receive (with or without baseline treatment) an SC injection of satralizumab at Weeks 0, 2, and 4, and Q4W thereafter, with the last study drug administration on or before 31 December 2021. |
| Name | Type | Description |
|---|---|---|
| Satralizumab | DRUG | Participants will receive satralizumab treatment for a minimum of 48 weeks and then will have the opportunity to enter an optional satralizumab extension (OSE) period. |
| Placebo | DRUG | Placebo will be administered by SC injection |
| azathioprine (AZA) | DRUG | Participants are permitted to use AZA during the study as background immunosuppressive treatment at a maximum dose of 3 milligram per kilogram per day (mg/kg/day) |
| mycophenolate mofetil (MMF) | DRUG | Participants are permitted to use MMF during the study as background immunosuppressive treatment at a maximum dose of 3000 mg/day |
| oral corticosteroids | DRUG | Participants are permitted to use oral corticosteroids (prednisolone equivalent) during the study as background immunosuppressive treatment at a maximum dose of 15 mg/day |
| Baseline Treatment | DRUG | As specified in the protocol, one of the following drugs at a stable dose is required as monotherapy for baseline treatment during the double-blind period: azathioprine (AZA); mycophenolate mofetil (MMF); or oral corticosteroids (CS). For participants aged 12 to 17 years at the time of informed consent, baseline treatment with AZA or MMF in combination with oral CS is also permitted. Change or termination of baseline treatment is only permitted during the open-label extension period. |
Inclusion Criteria: * Age at screening 2-11 years, inclusive * Body weight at screening \>=10 kg * For female patients of childbearing potential (postmenarchal): agreement to either remain completely abstinent (refrain from heterosexual intercourse) or to use a reliable means of contraception * Dia...
Satralizumab is an investigational monoclonal antibody being studied for several neurological and autoimmune conditions, including Thyroid Eye Disease, Neuromyelitis Optica, Neuromyelitis Optica Spectrum Disorder, Duchenne Muscular Dystrophy, NMDAR Autoimmune Encephalitis, and Myelin Oligodendrocyte Glycoprotein Antibody-Associated Disease (MOGAD). It is currently in Phase 2 clinical development.
Satralizumab is a monoclonal antibody, classified as a -mab, that targets the interleukin-6 (IL-6) receptor. By blocking this receptor, it is designed to modulate immune responses involved in autoimmune and inflammatory conditions. It is being investigated for diseases such as Thyroid Eye Disease and NMDAR Autoimmune Encephalitis.
Satralizumab is being developed by Roche Holding AG, a multinational healthcare company traded under the ticker RHHBY. Roche is conducting clinical trials to evaluate the drug's efficacy and safety across multiple indications, including Thyroid Eye Disease and Duchenne Muscular Dystrophy.
Satralizumab is currently in Phase 2 clinical development. While some of its trials are listed as Phase 3, the overall development stage for the drug is Phase 2. It is an investigational agent and has not been approved by regulatory authorities for any indication.
Satralizumab is being studied in several clinical trials. NCT05503264 is a Phase 3 trial in NMDAR and LGI1 autoimmune encephalitis, currently active but not recruiting. NCT05987423 and NCT06106828 are completed Phase 3 trials in Thyroid Eye Disease. NCT06450639 is a Phase 2 trial in Duchenne Muscular Dystrophy, also active but not recruiting.
Satralizumab is also known by the brand name Enspryng. It is a monoclonal antibody targeting the IL-6 receptor, developed by Roche. The drug is being investigated for conditions such as Neuromyelitis Optica Spectrum Disorder and Thyroid Eye Disease, though it remains in clinical development.