Evrysdi

Evrysdi, the brand name for risdiplam, is an oral medication approved for the treatment of spinal muscular atrophy (SMA). Developed by Roche, it is a small molecule that modulates the splicing of the SMN2 gene to increase the production of functional SMN protein, addressing the deficiency caused by mutations in the SMN1 gene.

Indications and usage

Evrysdi is indicated for the treatment of SMA in patients aged 2 months and older. It is suitable for various SMA types and is intended as a systemic therapy that can be taken at home.

Administration and dosing

Evrysdi is taken once daily as an oral solution. Dosing is weight- and age-based, and patients or caregivers should follow the prescription label and clinician instructions to ensure correct preparation and administration.

Mechanism of action

As an SMN2 splicing modifier, risdiplam promotes the production of full-length SMN protein from the SMN2 gene. By increasing SMN protein levels in the nervous system and other tissues, it aims to compensate for the loss of SMN1 function that underlies SMA.

Clinical evidence

Clinical trials have shown that risdiplam can improve motor function measures and enable some patients to achieve motor milestones compared with controls. Real-world outcomes vary, and long-term data are continually being collected to assess durability and broad applicability across SMA types and ages.

Safety and adverse effects

Common adverse events reported in trials include fever, rash, diarrhea, vomiting, and upper respiratory infections. Serious adverse events such as pneumonia can occur. As with any SMA therapy, safety monitoring and regular clinical assessments are recommended to manage potential risks.