SUNFISH Part 2: 24-Month Efficacy and Safety of Risdiplam in Patients with Type 2 or Non-Ambulant Type 3 Spinal Muscular Atrophy

Background/Purpose: Spinal muscular atrophy (SMA) is a severe, progressive neuromuscular disease caused by reduced levels of the survival of motor neuron (SMN) protein due to deletions/mutations of the SMN1 gene. Another gene, SMN2, produces only low levels of functional SMN protein. Risdiplam is an oral SMN2 pre-mRNA splicing modifier that increases levels of functional SMN protein and has been approved by the FDA for the treatment of patients with SMA, aged ≥2 months. This study's purpose is to determine the efficacy and safety of risdiplam in patients with Type 2 and non-ambulant Type 3 SMA after 24 months of treatment.

Methods: SUNFISH (NCT02908685) is a multicenter, two-part, randomized (2:1, risdiplam:placebo), placebo-controlled, double-blind study in a broad population of patients with types 2/3 SMA (inclusion criteria 2–25 years at enrollment). Part 2 assessed efficacy/safety of the Part 1-selected dose of risdiplam. Individuals were treated with risdiplam or placebo for 12 months, and then all received risdiplam until month 24. Afterward, patients had the opportunity to enter the open-label extension phase.

Results: The primary outcome of the study was met, showing a statistically significant difference in the change from baseline in MFM32 total score at month 12 between risdiplam (N = 120) and placebo (N = 60). No treatment-related safety findings leading to withdrawal were reported.

We will present efficacy/safety data after 24 months of treatment.

Conclusion: The ongoing SUNFISH part 2 will provide long-term efficacy and safety data of risdiplam in a broad population of children, teenagers and adults.

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Publikationsverlauf

Artikel online veröffentlicht:
28. Oktober 2021