24-month pooled FIREFISH data of risdiplam in SMA

In the phase 2/3 FIREFISH trial, infants with spinal muscular atrophy (SMA) treated with risdiplam continued to improve their ability to sit without support and other motor milestones after 24 months. Event-free survival time was greatly improved compared with natural history, while the rate of serious adverse events almost halved during the second 12-month period, compared with the first.

Risdiplam is approved for the treatment of patients with SMA aged ≥2 months. FIREFISH (NCT02913482) is an ongoing, multicentre, open-label study of risdiplam in infants with Type 1 SMA. Patients were aged 1–7 months at enrolment and had 2 SMN2 gene copies. Part 1 of the trial (n=21) assesses safety, tolerability, and pharmacokinetics/pharmacodynamics (PK/PD) of different risdiplam doses; part 2 of the trial (n=41) assesses efficacy and safety of risdiplam at the dose selected in part 1. Presented were the pooled safety and efficacy data of 58 participants in FIREFISH part 1 (high-dose cohort, n=17) and part 2 (n=41), who have been treated over 24 months [1].

Treatment over 24 months led to further improvement in the ability to sit without support. After 12 months of treatment, 19 of 58 infants (33%) could sit without support for ≥5 seconds; 11 of 58 (19%) could sit without support for ≥30 seconds. After 24 months of treatment, 35 of 58 (60%) could sit without support for ≥5 seconds; 23 of 58 (60%) for ≥30 seconds. Treatment also resulted in continued gains in motor milestones (HINE-2) after 24 months: 48 of 58 infants (83%) had treatment response using the HINE-2 scale and prespecified response criteria. Swallowing and feeding ability was maintained by the majority of infants alive at month 24. Event-free survival time was greatly improved in infants treated with risdiplam compared with historical data: 91% were alive after 24 months and 83% were event-free. The rate of hospitalisations and of serious adverse events both almost halved during the second 12-month period compared with the first 12 months.

1. Masson R, et al. FIREFISH Parts 1 and 2: 24-Month Safety and Efficacy of Risdiplam in Type 1 SMA. EPR-281, EAN 2021 Virtual Congress, 19–22 June.

 

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Posted on 18 August 202118 August 2021