Evrysdi is designed to address the underlying cause of SMA

The symptoms of SMA are caused by SMN protein deficits

SMA is caused by insufficient levels of functional SMN protein throughout the body²

Limited functional protein graphic

SMN protein deficiencies can have a widespread impact callout

People with SMA may face severe consequences

In infantile-onset disease, most patients are not expected to survive

<25% of patients are expected to survive but are not expected to sit independently graphic

In later-onset disease, children and adults may face progressive loss of function

Later-onset SMA patients may experience motor function impairment and limb weakness graphic

Proposed MOA

Evrysdi, an oral SMN2-splicing modifier, is designed to address the cause of SMA¹

Evrysdi™ is an SMN2-splicing modifier graphic

Based on preclinical studies, Evrysdi was shown to increase exon 7 inclusion in SMN2 mRNA transcripts¹
- 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

Animal models callout

Evrysdi led to an increase in SMN protein

In clinical trials, treatment increased and sustained functional SMN protein levels¹

A >2-fold increase in median SMN protein levels observed at 4 weeks graphic

SEE EVRYSDI STUDY DETAILS

CNS=central nervous system; MOA=mechanism of action; SMN=survival motor neuron.

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Indication

Evrysdi^™ (risdiplam) is indicated for the treatment of spinal muscular atrophy (SMA) in patients 2 months of age and older.

Interactions with Substrates of MATE Transporters

Based on in vitro data, Evrysdi may increase plasma concentrations of drugs eliminated via MATE1 or MATE2-K, such as metformin
Avoid coadministration of Evrysdi with MATE (multidrug and toxin extrusion) substrates. If coadministration cannot be avoided, monitor for drug-related toxicities and consider dosage reduction of the coadministered drug if needed

Pregnancy

In animal studies, administration of Evrysdi during pregnancy or throughout pregnancy and lactation resulted in adverse effects on development
Based on animal data, advise pregnant women of the potential risk to the fetus. Pregnancy testing is recommended for females of reproductive potential prior to initiating Evrysdi. Advise female patients of reproductive potential to use effective contraception during treatment with Evrysdi and for at least 1 month after the last dose

Breastfeeding

There is no data on the presence of Evrysdi in human milk, the effects on the breastfed infant, or the effects on milk production. The developmental and health benefits of breastfeeding should be considered along with the mother's clinical need for Evrysdi and any potential adverse effects on the breastfed infant

Potential Effects on Male Fertility

Male fertility may be compromised by treatment with Evrysdi. Counsel male patients on the potential effects on fertility. Male patients may consider sperm preservation prior to treatment

Hepatic Impairment

The safety and efficacy of Evrysdi in patients with hepatic impairment have not been studied
Because Evrysdi is predominantly metabolized in the liver, hepatic impairment may potentially increase the exposures to Evrysdi. Avoid use of Evrysdi in patients with impaired hepatic function

Most Common Adverse Reactions

The most common adverse reactions in later-onset SMA (incidence in at least 10% of patients treated with Evrysdi and more frequent than control) were fever, diarrhea, and rash
The most common adverse reactions in infantile-onset SMA were similar to those observed in later-onset SMA patients. Additionally, adverse reactions with an incidence of at least 10% were upper respiratory tract infection, pneumonia, constipation, and vomiting

You may report side effects to the FDA at 1-800-FDA-1088 or www.fda.gov/medwatch. You may also report side effects to Genentech at 1-888-835-2555.

Please see full Prescribing Information for additional Important Safety Information.