TABLE 2.

Efficacy and Safety of US Food and Drug Administration–Approved Therapies for NMOSD

	Pivotal studies	OLE results	OLE relapse rates	OLE AEs	OLE discontinuation	OLE EDSS scores
Eculizumab	PREVENT	96.1% of patients initially receiving eculizumab and 94.8% initially receiving placebo were relapse-free at 192 weeks96	The adjudicated ARR was 0.025 for the all eculizumab-treated patients96	Rates of treatment-related AEs and SAEs were 62% (183.5/100 PY) and 13.9% (8.6/100 PY), respectively96	11.8% of patients (14 of 119) discontinued treatment96	Mean EDSS scores maintained or continued to trend toward improvement96
Inebilizumab	N-MOmentum	87.7% of patients initially receiving inebilizumab and 83.4% initially receiving placebo remained attack-free for up to 4 years97	The adjudicated ARR was 0.052 for the patients with AQP4+ receiving inebilizumab98	Rates of treatment-related AEs and SAEs were 38.7% and 2.7%, respectively98	19.4% of patients (42 of 216) discontinued treatment99	Mean EDSS scores for patients with AQP4+ were stable throughout98
Satralizumab	SAkuraSky	71% of satralizumab-treated patients were relapse-free at 192 weeks100	The overall ARR was 0.11 from the first satralizumab dose to the cut-off date101	Rates of AEs and SAEs were 383.4/100 PY and 13.7/100 PY, respectively101	21.4% of patients (9 of 42) discontinued treatment102	90% had no sustained worsening of EDSS100
	SAkuraStar	73% of satralizumab-treated patients were relapse-free at 192 weeks100	The overall ARR was 0.08 from the first satralizumab dose to the cut-off date101	Rates of AEs and SAEs 324.8/100 PY and 10.6/100 PY, respectively101	18.2% of patients (6 of 33) discontinued treatment103	86% had no sustained worsening of EDSS100

AE = adverse event; ARR = adjudicated annualized relapse; EDSS = expanded disability status scale; NMOSD = neuromyelitis optica spectrum disorder; OLE = open label extension; PY = patient-year; SAE = serious adverse event.