Risdiplam · Evrysdi, RG-7916

What is Risdiplam?

Risdiplam is a small-molecule SMN2 splicing modifier that increases exon 7 inclusion in SMN2 mRNA transcripts, promoting full-length SMN protein production in the brain. The drug may also affect alternative splicing of FOXM1 and MADD genes.

Summary written with AI from the sources listed at the end of this page.

At a glance

Also known as

Evrysdi, RG-7916, RO-7034067

Drug type

Small molecule

Targets

SMN2, FOXM1 (secondary target), MADD (secondary target)

Mechanism

Modulator of SMN2

Pathways

alternative splicing, exon 7 inclusion

Therapy area

Neurology

Developer

Genentech Inc

In trials of

SRRK

FDA status

Approved as Evrysdi (August 7, 2020), NDA213535 · FDA label

UNII

76RS4S2ET1

✅ FDA-approved uses

• Spinal muscular atrophy

From the indications section of the FDA label (version of Feb 19, 2026). Read the full label for the exact wording, limitations and safety information.

🧪 Clinical trials

Trials of the companies BioRadar tracks, from ClinicalTrials.gov, most advanced phase first.

Conditions studied

Anti-myostatinNeuromuscular ManifestationsSMASpinal Muscular AtrophySpinal Muscular Atrophy Type 2Spinal Muscular Atrophy Type 3

Other drugs acting on SMN2

Sources

• FDA label (DailyMed)
• FDA label Evrysdi (2026-02-19), indications
• ClinicalTrials.gov NCT07047144

Updated Oct 3, 2026.