Efficacy and safety of risdiplam in patients with type 1 spinal muscular atrophy: a 3-year open-label extension of the two-part, phase 2 FIREFISH trial.

What was studied

FIREFISH was a phase 2 trial of risdiplam. The infants had type 1 spinal muscular atrophy and were aged 1-7 months. This report covers a 3-year open-label extension. The aim was to check long-term safety and effect. Children who finished 2 years of treatment could continue. The drug was given by mouth or feeding tube once daily. The study ran in 17 centres across 12 countries.

What they found

62 children were enrolled. 55 of them continued into the extension. After 5 years, 56 children were alive. 50 were alive and did not need permanent ventilation. By year 5, 36 children could sit unaided for 5 s or longer. The HINE-2 motor score rose up to year 4 and then held steady. CHOP-INTEND scores rose and settled around 50 by year 5. No child could walk alone. Most side effects were mild or moderate. Pneumonia was the most common serious side effect, in 28 children.

What it means for patients

The authors say most children were alive without permanent ventilation after 5 years. Most could swallow and feed by mouth. They had reached motor milestones not typically seen in untreated children. The authors say the results show lasting benefit and safety of risdiplam. No new safety findings were reported.

Why it is not certain yet

This was an open-label extension, so everyone knew the treatment. The abstract compares results with the usual disease course, not a control group. Results are reported at year 5.

Source

Efficacy and safety of risdiplam in patients with type 1 spinal muscular atrophy: a 3-year open-label extension of the two-part, phase 2 FIREFISH trial.

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