Last May 2023 marks four years since the FDA first approved a viral vector gene therapy, onasemnogene abeparvovec-xioi (ZOLGENSMA®), for treating pediatric patients with spinal muscular atrophy (SMA), a rare genetic condition characterized by the progressive loss of motor neurons leading to muscle weakness and atrophy. SMA is caused by a mutation in the survival motor neuron 1 (SMN1) gene that results in reduced or absence of the SMN protein.
While ZOLGENSMA® has shown significant benefits and is indicated for treating patients under two years of age, it is not a cure, and remaining unmet needs have been documented.
Besides ZOLGENSMA®, two other disease-modifying therapies are approved for treating SMA: Biogen/Ionis’s anti-sense oligonucleotide-based SPINRAZA® (nusinersen) and Roche/PTC’s small molecule-based splicing modulator EVYRSDI (risdiplam).
These three products represent a significant development in SMA treatment options since 2016. Current SMA drug development efforts focus on improving muscle functions and drug combinations to further improve patient outcomes.
Download this report to learn more about the emerging treatment options in SMA.