Clinical Trial and Postmarketing Safety of Onasemnogene Abeparvovec Therapy
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Why the study?
To comprehensively describe the safety data for intravenous onasemnogene abeparvovec across preclinical studies, clinical studies, and postmarketing sources.
What is the safety profile of intravenous onasemnogene abeparvovec in patients with spinal muscular atrophy?
Population
102 patients in clinical trials, 665 postmarketing adverse event cases, and preclinical animal models
Authors
Discussion
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Implication
Initial safety data for onasemnogene abeparvovec now available; leaves open long-term risk assessment in spinal muscular atrophy.
Structured PICO
What is the safety profile of intravenous onasemnogene abeparvovec in patients with spinal muscular atrophy?
P
Population
Patients with spinal muscular atrophy (clinical trials n=102, postmarketing n=665 adverse event cases) and preclinical models (neonatal mice, juvenile or neonatal cynomolgus nonhuman primates).
I
Intervention
Intravenous onasemnogene abeparvovec
O
Outcome
Safety (assessed through adverse event monitoring, vital-sign and cardiac assessments, laboratory evaluations, physical examinations, and concomitant medication use)safety
Intravenous onasemnogene abeparvovec therapy is associated with manageable safety risks, including hepatotoxicity that resolves with prednisolone, transient thrombocytopenia, and thrombotic microangiopathy.
Cite This Study
Mendell et al. (2021) studied this question.
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