Neuromyelitis optica spectrum disorder (NMOSD) is an autoimmune inflammatory disease of the central nervous system that primarily affects the optic nerves and spinal cord, often associated with AQP4-IgG antibodies.
Phase 3 trial data show daratumumab reduced relapse risk by 74% in NMOSD. Separately, the FDA accepted an NDA for iberdomide plus daratumumab/dexamethasone in relapsed/refractory multiple myeloma, with a PDUFA date of August 17, 2026.
Over 10 companies are developing 12+ pipeline therapies for Neuromyelitis Optica Spectrum Disorder, including Roche's satralizumab Phase III pediatric trial and multiple anti-CD20 monoclonal antibody candidates.
Amgen received FDA and European Commission approval for Uplizna to treat generalized myasthenia gravis in adults who are anti-AChR or anti-MuSK antibody positive, offering twice-yearly maintenance dosing after initial loading doses.
| NCT ID | Title | Status | Phase |
|---|---|---|---|
| NCT07557420 |
Efficacy, Safety, Pharmacokinetics, Pharmacodynamics, and Immunogenicity Study of Ravulizumab in Chinese Adults With Neuromyelitis Optica Spectrum Disorder (NMOSD) |
NOT_YET_RECRUITING | PHASE3 |
| NCT06561009 |
Safety and Efficacy of BAFFR CART for Relapsed/ Refractory Neuromyelitis Optica Spectrum Disorder |
NOT_YET_RECRUITING | PHASE1/PHASE2 |
| NCT06502015 |
Biomarkers in Autoimmune Disease of Nervous System |
RECRUITING | |
| NCT06497374 |
FcRn Antagonists (Efgartigimod) for Acute NMOSD Attack |
NOT_YET_RECRUITING | PHASE2 |
| NCT06474520 |
Efficacy and Safety of Calculus Bovis Sativus (CBS) for Idiopathic Inflammatory Demyelinating Disease (CBSinIIDD) |
RECRUITING | NA |
| NCT06374264 |
Acceptability and Safety of MR-C-014 in Persons With Neuromyelitis Optica Spectrum Disorder |
ENROLLING_BY_INVITATION | NA |
| NCT06180278 |
Long-term, Open-label, Safety Study of Inebilizumab in Neuromyelitis Optica Spectrum Disorder (NMOSD) |
ACTIVE_NOT_RECRUITING | PHASE4 |
| NCT05966467 |
Registry of Patients With AQP4+ NMOSD Treated With Alexion C5 Inhibitor Therapies |
RECRUITING | |
| NCT05909761 |
Observational Safety Study in Women With Neuromyelitis Optica Spectrum Disorder (NMOSD) Exposed to UPLIZNA® During Pregnancy |
RECRUITING | |
| NCT05891379 |
Inebilizumab in Acute Neuromyelitis Optica Spectrum Disorders |
RECRUITING |
