The symptoms of SMA are caused by SMN protein deficits
SMA is caused by insufficient levels of functional SMN protein throughout the body 2
People with SMA may face severe consequences
In infantile-onset disease, most patients are not expected to survive
In later-onset disease, children and adults may face progressive loss of function
Proposed MOA
Evrysdi, an oral SMN2-splicing modifier, is designed to address the cause of SMA 1
- Based on preclinical
studies, Evrysdi was shown to increase exon 7 inclusion in
SMN2 mRNA transcripts 1
- This led to an increased production of full-length
SMN protein in the brain
- Preclinical data also indicates Evrysdi may cause alternative splicing of other genes, including FOXM1 and MADD
- This led to an increased production of full-length
SMN protein in the brain
Evrysdi led to an increase in SMN protein
In clinical trials, treatment increased and sustained functional
SMN protein levels 1
CNS=central nervous system; MOA=mechanism of
action; SMN=survival motor neuron.