For Patients and Caregivers

NOW APPROVED: Evrysdi tablet formulation1

For illustrative purposes only.

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Extensively studied, with a consistent safety profile across all approved doses1

Extensively studied, with a consistent safety profile across all approved doses1

Patients living with SMA
SUNFISH PART 2
Adverse reactions occurring in ≥5% of adults and children receiving Evrysdi and with an incidence of ≥5% compared with placebo (N=180)1
  Evrysdi
(n=120)		 Placebo
(n=60)
Adverse reaction
Fever* 22% 17%
Diarrhea 17% 8%
Rash† 17% 2%
Mouth and aphthous ulcers 7% 0%
Arthralgia 5% 0%
Urinary tract infection‡ 5% 0%

As of clinical cut-off date: September 6, 2019.1

  • The most common adverse reactions reported in ≥10% of adults and children receiving Evrysdi and at an incidence greater than placebo were fever, diarrhea, and rash1

*Includes pyrexia and hyperpyrexia.

†Includes rash, erythema, rash maculopapular, rash erythematous, rash papular, dermatitis allergic, and folliculitis.1

‡Includes urinary tract infection and cystitis.1

SUNFISH PART 2
Safety observations over 4 years2,40

 

  Placebo
0-12 months
(n=60)*		 Evrysdi
0-12 months
(n=120)		 Evrysdi
12-24 months
(n=117)		 Evrysdi
24-36 months
(n=116)		 Evrysdi
36-48 months
(n=112)
Most common adverse events,† % (n)
Upper respiratory tract infection 30.0% (18) 31.7% (38) 16.2% (19) 8.6% (10) 17.9% (20)
Cold 25.0% (15) 25.8% (31) 22.2% (26) 9.5% (11) 5.4% (6)
Fever 16.7% (10) 20.8% (25) 13.7% (16) 9.5% (11) 8.0% (9)
Headache 16.7% (10) 20.0% (24) 10.3% (12) 7.8% (9) 3.6% (4)
Diarrhea 8.3% (5) 16.7% (20) 8.5% (10) 6.0% (7) 3.6% (4)
Vomiting 23.3% (14) 14.2% (17) 13.7% (16) 6.9% (8) 7.1% (8)
Cough 20.0% (12) 14.2% (17) 10.3% (12) 4.3% (5) 2.7% (3)
Most common serious adverse events, % (n)
Pneumonia 3.3% (2) 8.3% (10) 6.8% (8) 1.7% (2) 2.7% (3)
Influenza 0% (0) 0.8% (1) 0.9% (1) 0.9% (1) 0.9% (1)

Clinical cut-off date for Evrysdi 0-12 months and placebo 0-12 months: September 6, 2019.2,40

Clinical cut-off date for Evrysdi 12-24 months: September 30, 2020.2,40

Clinical cut-off date for Evrysdi 24-36 months: September 6, 2021.2,40

Clinical cut-off date for Evrysdi 36-48 months: September 6, 2022.2,40

*Patients in the placebo arm received placebo for 12 months followed by Evrysdi. The patients who started in the placebo group and switched to Evrysdi are not included in the 12- to 24-month and 36-month, and 36- to 48-month data.52

†Adverse reactions have an assumed causality to drug treatment under appropriate use; adverse events include any untoward event associated with the use of a drug, regardless of causality.1,53

  • No treatment-related adverse events leading to withdrawal or treatment discontinuation over 4 years40‡

‡One patient withdrew from the trial after the September 6, 2021 clinical cut-off date because of an adverse event (transaminitis) that was initially reported as related to Evrysdi, and then reassessed after discontinuation as unrelated to Evrysdi.54

The most common adverse reactions* to Evrysdi in infantile-onset SMA with an incidence of at least 10% were fever, diarrhea, rash, upper respiratory tract infection (including nasopharyngitis, rhinitis), lower respiratory tract infection (including pneumonia, bronchitis), constipation, vomiting, and cough1

FIREFISH PART 1 AND PART 2

Adverse events* occurring in ≥10% of infants receiving Evrysdi (N=62)2
All-patient cohort (N=62)

  Evrysdi
(N=62)
Adverse event
Upper respiratory tract infection (URTI)† 82%
Pyrexia 55%
Lower respiratory tract infections (LRTI)‡ 53%
Rash§ 29%
Constipation 26%
Diarrhea 19%
Vomiting 18%
Cough 13%
Teething 13%

As of clinical cut-off date: November 12, 2020.2

*Adverse reactions have an assumed causality to drug treatment; adverse events include any untoward event associated with the use of a drug, regardless of causality.53
†URTI includes COVID-19, influenza, nasopharyngitis, pharyngitis, respiratory tract infection, respiratory tract infection viral, rhinitis, upper respiratory tract infection, upper respiratory tract infection bacterial, viral upper respiratory tract infection.2
‡LRTI includes bronchiolitis, bronchitis, COVID-19, lower respiratory tract infection, lower respiratory tract infection viral, pneumonia, pneumonia aspiration, pneumonia bacterial, pneumonia staphylococcal, pneumonia viral, respiratory syncytial virus bronchiolitis, tracheitis, tracheobronchitis.2
§Rash includes rash, dermatitis, dermatitis acneiform, dermatitis allergic, erythema, folliculitis, rash maculopapular, rash papular.2

  • No treatment-related adverse events leading to treatment discontinuation over 2 years55

As of clinical cut-off date: November 12, 2020.

  • The safety findings from JEWELFISH have been consistent with the safety findings in FIREFISH and SUNFISH33
RAINBOWFISH
In infants from the ITT population

 

  • 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 rash1
  • 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 cough1
  • Adverse events observed in presymptomatic SMA were consistent with those observed in infantile-onset and later-onset SMA1
  • No treatment-related adverse events leading to discontinuation41

As of clinical cut-off date: February 20, 2023.

Most common adverse events occurring in ≥5 infants receiving Evrysdi41
  Evrysdi
(N=26)
Adverse events
Teething 39%
COVID-19 35%
Pyrexia 27%
Gastroenteritis 27%
Eczema 23%
Constipation 23%
Diarrhea 23%
Nasopharyngitis 19%
Rhinitis 19%
Vomiting 19%
Nasal congestion 19%

As of clinical cut-off date: February 20, 2023.

  • Multiple occurrences of the same adverse event in an individual are counted only once41
  • Includes adverse events with onset from first dose to the cut-off date41

*Adverse reactions have an assumed causality to drug treatment under appropriate use; adverse events include any untoward event associated with the use of a drug, regardless of causality.53

ITT=intent-to-treat.

PREVIOUS TREATMENT
JEWELFISH adverse events and serious adverse events at 2 years
  RG7800*
(MOONFISH)(n=13)†		 Nusinersen
(n=76)		 Olesoxime*
(n=71)		 Onasemnogene abeparvovec
(n=14)		 All patients
(N=174)‡
Most common adverse events (reported in ≥12% of all patients), % (n)
Pyrexia 15% (2) 30% (23) 16% (11) 43% (6) 24% (42)
Upper respiratory tract infection 0 22% (17) 23% (16) 29% (4) 21% (37)
Headache 8% (1) 22% (17) 19% (13) 0 18% (31)
Nasopharyngitis 15% (2) 16% (12) 11% (8) 36% (5) 16% (27)
Diarrhea 0 22% (17) 9% (6) 7% (1) 14% (24)
Nausea 0 18% (14) 10% (7) 7% (1) 13% (22)
Cough 0 16% (12) 9% (6) 21% (3) 12% (21)
Most common serious adverse events (reported in >2% of all patients),§ % (n)
Pneumonia 0 4% (3) 1% (1) 7% (1) 3% (5)
Respiratory failure 0 4% (3) 1% (1) 0 2% (4)
Respiratory distress 0 3% (2) 0 7% (1) 2% (3)
Lower respiratory tract infection 0 1% (1) 3% (2) 0 2% (3)
Upper respiratory tract infection 0 4% (3) 0 0 2% (3)

As of clinical cut-off date: January 31, 2022.33

*Investigational therapies; not approved by FDA for any use.47

†In the MOONFISH trial, 3 patients were treated with placebo and were not switched to RG7800.33

‡One patient withdrew from the study at baseline; therefore, 173 patients received Evrysdi.33

§Multiple occurrences of the same adverse event in one individual are counted only once.33

  • Evrysdi treatment duration in months, median (range):  26.8 (0.9-59.0)33
  • As follow-up duration is different between groups, the overall rate of adverse events and serious adverse events cannot be compared33
  • The safety findings from JEWELFISH have been consistent with the safety findings in FIREFISH and SUNFISH33
No required monitoring or laboratory testing1

When initiating Evrysdi, it is important to be aware of any medications your patient is currently taking. This includes prescription medications (such as statins), over-the-counter medications (such as stool softeners), vitamins, dietary supplements, or additional fiber that may impact bowel movements.

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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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    • FDA approves Genentech’s Evrysdi (risdiplam) for treatment of spinal muscular atrophy (SMA) in adults and children 2 months and older. News Release. Genentech USA, Inc.; October 3, 2020. Accessed October 3, 2024. https://www.gene.com/media/press-releases/14866/2020-08-07/fda-approves-genentechs-evrysdi-risdiplam

      FDA approves Genentech’s Evrysdi (risdiplam) for treatment of spinal muscular atrophy (SMA) in adults and children 2 months and older. News Release. Genentech USA, Inc.; October 3, 2020. Accessed October 3, 2024. https://www.gene.com/media/press-releases/14866/2020-08-07/fda-approves-genentechs-evrysdi-risdiplam

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