The European Commission (EC) has approved label expansion for Roche’s Evrysdi (risdiplam) in spinal muscular atrophy (SMA) to include patients aged two months and under.

This builds upon Evrysdi’s existing European approval for patients aged two months and older and echoes a US Food and Drug Administration (FDA) approval for the same group in May.

The drug was first approved for children and adults with SMA by the EC and FDA in 2021 and 2022, respectively.

Evrysdi is a survival motor neuron 2 (SMN2) splicing modifier that targets mutations in chromosome 5q that lead to survival motor neuron (SMN) protein deficiency. It is approved for the treatment of SMA Type I, Type II or Type III or with one to four SMN2 copies.

Roche reported sales of Sfr 705m ($801.4m) for Evrysdi in H1 2023, as per the company’s half-year report. Its main competitor Biogen’s Spinraza (nusinersen) had reported sales of $880.4m in the same period. However, GlobalData forecasts Evrysdi sales to eclipse Spinraza sales in 2029, with $2.98bn in global sales for Evrysdi compared to $1.3bn for Spinraza. The shift in sales is mainly due to the favourable oral delivery of Evrysdi compared to the intrathecal route (via a lumbar puncture) for Spinraza.

GlobalData is the parent company of Clinical Trial Arena.

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Evrysdi is being developed in collaboration with the SMA Foundation and PTC Therapeutics. The drug can be administered at home as a daily treatment orally or via a feeding tube.

Evrysdi approval data

Evrysdi’s approval was based on the interim data from the open-label Phase II trial (NCT03779334) from genetically diagnosed and pre-symptomatic Type I SMA patients from birth to six weeks of age.

All six infants included in the analysis were able to sit and breathe without permanent ventilation following a year of Evrysdi treatment. Of the six, four were able to stand and three were able to walk independently after a year.

The commonly observed side effects included fever, diarrhoea, rash, respiratory tract infection, constipation, and vomiting.

Evrysdi is also being evaluated as a combination therapy with an experimental anti-myostatin molecule, GYM329 (RO7204239), in a Phase II/III trial (NCT05115110) in SMA patients aged two to ten years.