For Patients and Caregivers

Results in newborns with presymptomatic SMA

Results in newborns with presymptomatic SMA

Evrysdi®️ (risdiplam) newborns living with SMA

RAINBOWFISH is an ongoing, open-label trial of Evrysdi in 26 newborns who were aged 16 to 40 days at first Evrysdi dose. These infants had a genetic diagnosis of SMA, but had not yet presented with symptoms (presymptomatic SMA). Six infants who had 2 or 3 copies of SMN2 were included in an interim efficacy analysis.1,28

Trial design

Newborns demonstrated meaningful function1,28

Evrysdi helped infants sit, stand, and walk

RAINBOWFISH

AFTER 12 MONTHS OF TREATMENT
n=6*

Infant sitting up
100% of infants

of infants (6/6) were able to sit without support, as measured by HINE-2

  • 5 infants could pivot/rotate
  • 1 infant achieved stable sit
Infant standing up
67% of infants

of infants (4/6) were able to stand, as measured by HINE-2

  • 3 infants could stand unaided
  • 1 infant could stand with support
Infant walking independently
50% of infants

of infants (3/6) were able to walk independently, as measured by HINE-2

  • 3 infants could walk independently

As of clinical cut-off date: July 1, 2021.

*Includes infants with 2 or 3 copies of SMN2.

HINE-2=Hammersmith Infant Neurological Examination–Module 2.

The Hammersmith Infant Neurological Examination–Module 2 (HINE-2) assesses 8 developmental milestones for infants, including head control, sitting, voluntary grasp, ability to kick, rolling, crawling, standing, and walking.

Evrysdi helped infants survive1

RAINBOWFISH

AFTER 12 MONTHS OF TREATMENT
n=6*

Breath without permanent support
100% of infants

of infants (6/6) were alive without permanent ventilation†

As of clinical cut-off date July 1, 2021.

*Includes infants with 2 or 3 copies of SMN2.

†Defined as requiring a tracheostomy or >21 consecutive days of either noninvasive ventilation (≥16 hours per day) or intubation, in the absence of an acute reversible event.

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Important Safety Information and Indication

Indication

Evrysdi is indicated for the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients.

 

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 & Breastfeeding

  • Evrysdi may cause embryofetal harm when administered to a pregnant woman. In animal studies, administration of Evrysdi during pregnancy and/or lactation resulted in adverse effects on development. Advise pregnant women of the potential risk to the fetus
  • Pregnancy testing is recommended prior to initiating Evrysdi. Advise female patients to use contraception during treatment with Evrysdi and for at least 1 month after the last dose
  • There is a pregnancy exposure registry that monitors pregnancy and fetal/neonatal/infant outcomes in women exposed to Evrysdi during pregnancy. Physicians are encouraged to register patients and pregnant women are encouraged to register themselves by calling 1-833-760-1098 or visiting www.evrysdipregnancyregistry.com
  • 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

  • Counsel male patients that fertility may be compromised by treatment with Evrysdi. Male patients may consider sperm preservation prior to treatment

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 (including nasopharyngitis, rhinitis), lower respiratory tract infection (including pneumonia, bronchitis), constipation, vomiting, and cough
  • The safety profile for presymptomatic patients is consistent with the safety profile for symptomatic SMA patients treated with Evrysdi in clinical trials

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.

 

 

    • Evrysdi® (risdiplam) Prescribing Information. Genentech, Inc.

      Evrysdi® (risdiplam) Prescribing Information. Genentech, Inc.

    • Data on file. Genentech USA, Inc.

      Data on file. Genentech USA, Inc.

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