Why carry out this study?

Spinal muscular atrophy (SMA) is a genetic disease that affects infants to adults with varying degrees of disease severity.

With disease-modifying therapies available for SMA, it is imperative to understand the safety of these drugs when used successively or in combination.

The aim of this interim analysis was to determine the safety and pharmacodynamics of risdiplam in a heterogeneous population of patients with SMA who had been previously enrolled in an SMA clinical trial or treated with a previously approved SMA treatment.

What was learned from the study?

The JEWELFISH study population had a similar safety profile and increase in SMN protein levels after 12 months of treatment with risdiplam compared with treatment-naïve patients who were treated with risdiplam in the FIREFISH and SUNFISH clinical trials.

No safety signals were observed in the patient population after 12 months of treatment with risdiplam.

There were no deaths or drug-related safety findings that led to the withdrawal of any patients treated with risdiplam.