Roche presented positive results from the Phase II Rainbowfish trial of Evrysdi (risdiplam) at the 28th World Muscle Society (WMS) Congress in Charleston (USA) from 3-7 October 2023.

The data presented includes the primary one-year analysis of the Rainbowfish trial in babies with pre-symptomatic spinal muscular atrophy (SMA), aged from birth to six weeks.

Evrysdi is a survival motor neuron 2 (SMN2) splicing modifier that targets mutations in chromosome 5q, which is implicated in survival motor neuron (SMN) protein deficiency.

It was approved to treat SMA in babies aged below two months by the US Food and Drug Administration (FDA) in 2022. The approval is for the treatment of SMA Type 1, Type 2 or Type 3 or with one to four SMN2 copies in all age groups.

Roche reported sales of Sfr 705m ($801.4m) for Evrysdi in H1 2023, as per the company’s half-year report. GlobalData forecasts Evrysdi sales to increase to $2.98bn in 2029.

GlobalData is the parent company of Clinical Trials Arena.

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Clinical trial data

The open-label Rainbowfish trial (NCT03779334) met its primary endpoint with 80% of the primary efficacy population sitting without support for at least five seconds after one year of Evrysdi treatment, assessed by Bayley Scales of Infant and Toddler Development, third edition (BSID-III).

Of the 26 babies in the study, 81% could sit independently for 30 seconds. Adverse events (AEs) for the drug included teething, Covid-19, fever, eczema, and constipation. However, the company was quick to add that the majority of AEs were not considered treatment-related.

Other trials of Evrysdi include two Phase II/III studies which met their primary endpoints, including the Firefish trial (NCT02913482) in babies with type 1 SMA, and the Phase II/III Sunfish trial (NCT02908685) in people aged 2-25 years with Types 2 or 3 SMA.

The continuing studies for Evrysdi monotherapy include Phase II Jewelfish trial (NCT03032172) in people with SMA aged 6 months to 60 years who received other investigational or approved SMA therapies for at least 90 days prior to receiving Evrysdi.

Evrysdi is also being evaluated as a combination therapy with an anti-myostatin molecule, GYM329 (RG6237), in SMA patients aged 2-10 years in the Phase II/III Manatee trial (NCT05115110).