Treatment with nusinersen improved motor function in infantile-onset SMA for up to 5 years

According to a poster presented at the International Scientific Conference on Spinal Muscular Atrophy, treatment with nusinersen led to sustained and improved motor function up to five years after the first dose in infants with spinal muscular atrophy.

“Long-term follow-up in the SHINE study will allow us to better understand emerging phenotypes in SMA.” Diana Castro, M.D. “In the absence of well-designed and controlled studies, physicians cannot advise new patients and their families on what to expect and what is realistic as they navigate life with SMA. You can not.”

Castro et al. sought to investigate the safety and efficacy of the antisense oligonucleotide nusinersen in adults and children with infantile-onset spinal muscular atrophy that transitioned from the ENDEAR trial to the SHINE trial. ENDEAR is a sham-controlled Phase 3 trial of nusinersen for up to 394 days in symptomatic infantile-onset patients with probable SMA Type 1 and SHINE is an ongoing, open-label extension study .

A total of 105 participants (80 started on ENDEAR and 25 on SHINE) received nusinersen in the SHINE trial, with a median duration of 5.3 years. Participants were analyzed in three groups according to age at first study dose and timing of first dose. Age 6 months to less than 10 months with previous nusinersen or sham surgery at ENDEAR (n = 29). From 10 months of age he was less than 23 months old and started nusinersen on SHINE after receiving her sham on ENDEAR (n = 25).

According to the results, the Philadelphia Children’s Hospital mean neuromuscular infant test scores from baseline to day 1,538 for all age groups (<6 months, 27 to 48.3; 6 months to <10 months, 25.9); from 43.8, from 10 months to less than 10 months). At 23 months (17.4 to 25.2), modified upper extremity module and Hammersmith Functional Motor Scale Expanded scores also continued to improve within the two youngest groups from baseline to 1,080 days.

Additionally, no serious adverse events were considered treatment-related as of the data cutoff date. The most common adverse events were fever, pneumonia, and upper respiratory tract infection, and the most common serious adverse events included pneumonia, dyspnea, and respiratory failure.

“The results from the SHINE trial show that patients continue to gain motor function, reach milestones after an average of five years of treatment, and have no significant side effects at up to seven years of follow-up,” said Castro. “With any therapy, it’s important to be able to continue to look forward to new motor milestones, even if they’re delayed.”