Advances & More News / Reported from USA: Five-Year Extension Results of the Phase 1 START Trial of Onasemnogene Abeparvovec in Spinal Muscular Atrophy

Spinal muscular atrophy (SMA) is a neuromuscular disorder represented by dysfunction and loss of alpha motor neurons in the spinal cord; consequently, patients with (SMA) usually suffer from progressive muscle atrophy, weakness, and paralysis. Onasemnogene abeparvovec is a recombinant adeno-associated virus serotype nine vector-based gene therapy available as a single intravenous infusion to deliver a full-length functional copy of the human SMN gene through a self-complementary adeno-associated virus serotype nine vectors which crosses the blood-brain barrier. An ongoing, observational, follow-up study published in JAMA Neurology aimed to evaluate the safety of Onasemnogene abeparvovec as a primary objective by assessing the incidence of side effects and determining if developmental milestones attained in the START phase 1 clinical trial and if new milestones are achieved as a secondary objective. The study's participants are symptomatic infants with SMA type 1, and 2 copies of SMN2 treated with an intravenous dose of onasemnogene abeparvovec (n=13). Results show that serious adverse events were reported in 8 patients; however, none of these events led to study discontinuation or death. The most prevalent adverse effects reported were an acute respiratory failure, dehydration, respiratory distress, and bronchiolitis. However, patients who received the therapeutic dose of Onasemnogene abeparvovec remained alive and didn't need permanent ventilation. Before reaching the baseline, four patients receiving the therapeutic dose needed noninvasive ventilatory support, and six did not, which didn't change in the long-term follow-up. Overall, all ten patients who were treated with the therapeutic dose maintained previously acquired motor milestones. On the other hand, two patients attained the new milestone of "standing with assistance" without the receiving nusinersen. In conclusion, study findings support the long-term favorable safety profile for SMA patients for up to 6 years providing evidence for sustained clinical durability of the therapeutic dose. By: Advances and More Medical Reporters Reference: Mendell, J. R., Al-Zaidy, S. A., Lehman, K. J., McColly, M., Lowes, L. P., Alfano, L. N., Reash, N. F., Iammarino, M. A., Church, K. R., Kleyn, A., Meriggioli, M. N., & Shell, R. (2021). Five-Year Extension Results of the Phase 1 START Trial of Onasemnogene Abeparvovec in Spinal Muscular Atrophy. JAMA neurology, 78(7), 834–841. https://doi.org/10.1001/jamaneurol.2021.1272
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