ABSTRACT
Introduction
Onasemnogene abeparvovec is the first systemic gene replacement therapy approved by the FDA for any inherited condition and is the second FDA-approved genetic therapy for 5q spinal muscular atrophy.
Areas covered
We discuss the design and preclinical development of onasemnogene abeparvovec, along with clinical trial and real-world data focusing on efficacy and safety.
Expert Opinion
Although onasemnogene abeparvovec is strikingly effective for the treatment of 5q SMA, it is only approved for use under the age of 2 years and there are also two other FDA-approved molecular-based treatments. Many questions remain in terms of treatment selection, possibility of dose optimization, and combinational therapies.
Article highlights
Onasemnogene abeparvovec is a systemic gene replacement therapy for spinal muscular atrophy using a viral vector, AA9
Onasemnogene abeparvovec is safe with appropriate prescreening and follow up for the treatment of spinal muscular atrophy
Onasemnogene abeparvovec is most effective when treatment is initiated prior to symptom onset
Even if given when some symptoms are present, onasemnogene abeparvovec can still be efficacious
Data on long-term safety and its use in combination with other spinal muscular atrophy genetic therapies will be areas of continued research