For Patients and Caregivers

RAINBOWFISH

Milestones that matter for newborns with presymptomatic SMA1

RAINBOWFISH is an open-label trial of Evrysdi in 26 newborns who were aged 16 to 41 days at first Evrysdi dose. These infants had a genetic diagnosis of spinal muscular atrophy (SMA), but had not yet presented with symptoms.1

Trial design

Early treatment with Evrysdi helped infants sit without support1

Results were clinically meaningful across populations, including the group with the more severe disease
RAINBOWFISH
AFTER 1 YEAR OF TREATMENT

INFANTS IN PRIMARY ANALYSIS POPULATION (n=5)

of infants (4/5)
were able to sit without support for at least 5 seconds
As measured by BSID-III, Item 22

INFANTS WITH MORE SEVERE DISEASE (n=8)

of infants (7/8)
were able to sit without support for at least 5 seconds
As measured by BSID-III, Item 22

Based on the natural history of infantile-onset Type 1 SMA, untreated infants never acquire the ability to sit without support41*

*According to a study of SMA patients, approximately 79% of patients with 2 SMN2 copies develop Type 1 SMA.41

As of clinical cut-off date: February 20, 2023.5

INFANTS IN ITT POPULATION (N=26)

of infants (25/26)
were able to sit without support for at least 5 seconds
As measured by BSID-III, Item 22

  • 81% of infants (21/26) were able to sit without support for 30 seconds

As measured by BSID-III, Item 26

Based on the natural history of infantile-onset Type 1 SMA, untreated infants never acquire the ability to sit without support41*

*According to a study of SMA patients, approximately 79% of patients with 2 SMN2 copies develop Type 1 SMA.41

As of clinical cut-off date: February 20, 2023.5

The Bayley Scales of Infant and Toddler Developmental-Third Edition (BSID-III) gross motor scale assesses a range of physical abilities, such as sitting, rolling, and crawling.1,25

EXPLORATORY ASSESSMENTS SUGGEST

Infants achieved a key sitting milestone after 2 years of taking Evrysdi42

RAINBOWFISH
AFTER 2 YEARS OF TREATMENT

INFANTS IN PRIMARY ANALYSIS POPULATION (n=4)*

of infants (4/4)
were able to sit without support for at least 5 seconds

of infants (4/4)
were able to sit without support for at least 5 seconds
As measured by BSID-III, Item 22

INFANTS WITH MORE SEVERE DISEASE (n=5)†

of infants (5/5)
were able to sit without support for at least 5 seconds

of infants (5/5)
were able to sit without support for at least 5 seconds
As measured by BSID-III, Item 22

INFANTS IN ITT POPULATION (N=23)†

of infants (23/23)
were able to sit without support for at least 5 seconds

of infants (23/23)
were able to sit without support for at least 5 seconds
As measured by BSID-III, Item 22

As measured by BSID-III, Item 22

As of clinical cut-off date: March 27, 2024.42

These data reflect the impact of Evrysdi in conjunction with the natural development toward achieving these milestones. Two-year assessments should be interpreted with caution, as these are exploratory data.

*Excludes 1 infant who withdrew before the Year 2 assessment to receive a one-time disease-modifying treatment.42
†Excludes 3 infants who withdrew before the Year 2 assessment to receive a one-time disease-modifying treatment.42

EXPLORATORY ASSESSMENTS SUGGEST

Majority of infants taking Evrysdi were able to sit without support for 30 seconds42

RAINBOWFISH
AFTER 2 YEARS OF TREATMENT

INFANTS IN PRIMARY ANALYSIS POPULATION (n=4)*

of infants (4/4)
were able to sit without support for 30 seconds

of infants (4/4)
were able to sit without support for 30 seconds
As measured by BSID-III-Item 26

INFANTS WITH MORE SEVERE DISEASE (n=5)†

of infants (5/5)
were able to sit without support for 30 seconds

of infants (5/5)
were able to sit without support for 30 seconds
As measured by BSID-III-Item 26

INFANTS IN ITT POPULATION (N=23)†‡

of infants (21/23)
were able to sit without support for 30 seconds

of infants (21/23)
were able to sit without support for 30 seconds
As measured by BSID-III-Item 26

As measured by BSID-III, Item 26

As of clinical cut-off date: March 27, 2024.42

These data reflect the impact of Evrysdi in conjunction with the natural development toward achieving these milestones. Two-year assessments should be interpreted with caution, as these are exploratory data.

*Excludes 1 infant who withdrew before the Year 2 assessment to receive a one-time disease-modifying treatment.42
†Excludes 3 infants who withdrew before the Year 2 assessment to receive a one-time disease-modifying treatment.42
‡For infants with 3 SMN2 copies, the clinical site evaluator results differed from the 2 independent central readers. The clinical site evaluator results were 13/13 infants were able to sit without support for 30 seconds, while the 2 independent central readers’ results were 12/13 infants. For infants with ≥4 SMN2 copies, the clinical site evaluator results differed from the 2 independent central readers. The clinical site evaluator results were 5/5 infants were able to sit without support for 30 seconds, while the 2 independent central readers’ results were 4/5 infants. The 2 independent reader results are reported here.42

IN INFANTS FROM THE ITT POPULATION

Evrysdi helped infants sit, stand, or walk independently1

RAINBOWFISH

AFTER 1 YEAR OF TREATMENT
(N=26)

of infants (24/25)*
were able to sit without support

of infants (21/25)*
were able to stand with and without support

  • 13/25 could stand unaided
  • 8/25 could stand with support

of infants (12/25)*
were able to walk independently

As measured by HINE-2

of infants (24/25)*
were able to sit without support
 As measured by HINE-2

of infants (21/25)*
were able to stand with and without support

  • 13/25 could stand unaided
  • 8/25 could stand with support

As measured by HINE-2

of infants (12/25)*
were able to walk independently
As measured by HINE-2

As of clinical cut-off date: February 20, 2023.5

*One infant with ≥4 SMN2 copies could not be assessed.5

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.1,40

EXPLORATORY ASSESSMENTS IN ITT POPULATION SUGGEST

Continued treatment helped infants hit motor milestones42

RAINBOWFISH

AFTER 2 YEARS OF TREATMENT
(N=23)*

of infants (23/23)
were able to sit without support

of infants (22/23)
were able to stand with and without support

  • 21/23 could stand unaided
  • 1/23 could stand with support

of infants (20/23)†
were able to walk independently

of infants (23/23)
were able to sit without support

of infants (22/23)
were able to stand with and without support

  • 21/23 could stand unaided
  • 1/23 could stand with support

of infants (20/23)†
were able to walk independently

As measured by HINE-2

As of clinical cut-off date: March 27, 2024.42

These data reflect the impact of Evrysdi in conjunction with the natural development toward achieving these milestones. HINE-2 assessments should be interpreted with caution, as these are exploratory data.

*Excludes 3 infants who withdrew before the Year 2 assessment to receive a one-time disease-modifying treatment.42
†One child could not be assessed at Year 2.42

IN INFANTS FROM THE ITT POPULATION

All infants taking Evrysdi survived without permanent ventilation after 1 and 2 years1,42*

RAINBOWFISH
AFTER 1 YEAR OF TREATMENT
(N=26)†
100%

of infants (26/26)
were alive without permanent ventilation1,13

As of clinical cut-off date: February 20, 2023.13

EXPLORATORY ASSESSMENTS SUGGEST AFTER 2 YEARS OF TREATMENT (N=23)‡

100% of infants (23/23) were alive without permanent ventilation42

Alive without permanent ventilation data should be interpreted with caution, as these are exploratory data.

As of clinical cut-off date: March 27, 2024.42

*Permanent ventilation was defined as a tracheostomy or >21 consecutive days of either non-invasive ventilation (≥16 hours per day) or intubation, in the absence of an acute reversible event.42
†8 patients had 2 SMN2 copies, 13 patients had 3 SMN2 copies, and 5 patients had ≥4 SMN2 copies.1
‡Excludes 3 infants who withdrew before the Year 2 assessment to receive a one-time disease-modifying treatment.42

EXPLORATORY ASSESSMENTS IN ITT POPULATION SUGGEST

All infants taking Evrysdi were able to feed exclusively by mouth and swallow42*

RAINBOWFISH
AFTER 2 YEARS OF TREATMENT
(N=23)†

of infants (23/23)
were able to feed exclusively by mouth

of infants (23/23)
were able to swallow

As of clinical cut-off date: March 27, 2024.42

Feeding and swallowing assessments should be interpreted with caution, as these are exploratory data.

*Assessed using nutritional status interview of the parent/caregiver and a standard swallowing assessment based on local practice and performed by a qualified individual. Swallowing assessment was completed at baseline and during follow-up visits. Ability of the patient to swallow age-appropriate foods was assessed.43
†Excludes 3 infants who withdrew before the Year 2 assessment to receive a one-time disease-modifying treatment. The 3 infants were able to swallow and feed orally at their last assessments.42

An open-label, single-arm trial1,42

Evaluating the efficacy and safety of Evrysdi in newborns with genetically diagnosed, presymptomatic SMA

  • Infants were <6 weeks of age at time of first Evrysdi dose, had a genetic diagnosis of SMA, but had not yet presented with symptoms1
  • The primary endpoint was the ability to sit without support for ≥5 seconds at 1 year, as measured by Item 22 of the BSID-III gross motor scale1

Efficacy was evaluated in 3 groups of infants that varied in disease severity at baseline (as measured by SMN2 copy number and CMAP amplitude)1

5 infants
(primary analysis population)1,5

  • 2 SMN2 copies
  • CMAP at baseline ≥1.5 mV
    (1.6-3.8 mV)

8 infants
(more severe disease)1,5

  • 2 SMN2 copies
  • CMAP at baseline 0.5-3.8 mV
    • Includes 3 infants with CMAP <1.5 mV at baseline
  • Includes the 5 infants from the primary analysis

26 infants
(intent-to-treat)1,5

  • 2 to ≥4 SMN2 copies
  • CMAP at baseline 0.5-6.7 mV

Representing a range of disease severity in presymptomatic SMA1,5

Infants had varied numbers of SMN2 copies and CMAP amplitudes

RAINBOWFISH
  Primary analysis population
(n=5)		 More severe disease
(n=8)		 ITT
(N=26)
Patient characteristics5
Age at first dose (days), median (range) 24.0 (22.0-35.0) 23.5 (16.0-35.0) 25.0 (16.0-41.0)
SMN2 copy number, % (n)
2
3
≥4
100.0% (5)
0.0% (0)
0.0% (0)
100.0% (8)
0.0% (0)
0.0% (0)
30.8% (8)
50.0% (13)
19.2% (5)
Gender, % (n)
Female
Male
60.0% (3)
40.0% (2)
50.0% (4)
50.0% (4)
61.5% (16)
38.5% (10)
Weight (g), median (range) 4045.0
(3865-4270)		 3999.0
(3076-4270)		 4015.0
(3076-5726)
Baseline CMAP amplitude (mV), median (range)
Baseline value <1.5 mV, % (n)
Baseline value ≥1.5 mV, % (n)

2.6 (1.6-3.8)

0.0% (0)
100.0% (5)

2.0 (0.5-3.8)

37.5% (3)
62.5% (5)

3.6 (0.5-6.7)

11.5% (3)
88.5% (23)

As of clinical cut-off-date: February 20, 2023.5

The only trial of an FDA-approved DMT that studied infants with presymptomatic SMA who had CMAP <1 mV1,5,44,45

BSID-III=Bayley Scales of Infant and Toddler Development-Third Edition; CMAP=compound muscle action potential; DMT=disease-modifying treatment; FDA=Food and Drug Administration; HINE-2=Hammersmith Infant Neurological Examination-Module 2; ITT=intent-to-treat; mV=millivolt; SMN2=survival motor neuron 2.

Understanding Evrysdi®️ and SMA support brochure icon

Overview of results from our clinical trial program

Download

Administering and storing Evrysdi

Non-invasive options to fit your patients’ needs1,21

See more

How Evrysdi works

Increasing SMN protein production throughout the body1

Learn more

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 https://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.

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    • Data on file. Genentech USA, Inc.

      Data on file. Genentech USA, Inc.

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      Mercuri E, Deconinck N, Mazzone ES, et al; on behalf of the SUNFISH Study Group. Safety and efficacy of once-daily risdiplam in type 2 and non-ambulant type 3 spinal muscular atrophy (SUNFISH Part 2): a phase 3, double-blind, randomised, placebo-controlled trial. Lancet Neurol. 2021;21(1)(suppl 1):42-52.

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