In Part 1 of FIREFISH—the study of patients with Type 1 SMA

Infants achieved the ability to sit without support for at least 5 seconds 1

This important motor milestone was measured by BSID-III

Evrysdi helped infants survive without permanent ventilation 1

Results after at least 12 months of treatment demonstrated efficacy during a critical time for infants with SMA

FIREFISH was a 2-part, multicenter, open-label trial that investigated the efficacy, safety, pharmacokinetics, and pharmacodynamics of Evrysdi in infants between 2 and 7 months of age at the time of enrollment, diagnosed with Type 1 SMA. Part 1 was the dose-finding portion in 21 infants; efficacy and safety were also evaluated on the ability to sit without support for at least 5 seconds as measured by Item 22 of the BSID–III gross motor scale and on the basis of survival without permanent ventilation. After the 12-month time point, all participants were treated with the daily, recommended dose (0.2 mg/kg).

 

In Part 2 of SUNFISH—the study of patients with Type 2 or Type 3 SMA

Evrysdi demonstrated efficacy in children and adults with varying levels of disease severity1

Evrysdi met its primary endpoint with significantly greater change in motor function score vs placebo at Month 12, as measured by MFM-32

  • 1.55-point difference (95% CI: 0.30, 2.81) between the means (P=0.0156)
  • The MFM-32 is a 32-item scale designed to assess various motor functions in people with neuromuscular disorders. The scale measures motor function abilities that relate to important daily functions 2,3

SUNFISH was a 2-part, multicenter trial that investigated the efficacy, safety, pharmacokinetics, and pharmacodynamics of Evrysdi in children and adults between 2 and 25 years of age, diagnosed with Type 2 or Type 3 SMA. Part 2 was the randomized, double-blind, placebo-controlled portion in 180 nonambulatory patients with Type 2 (71%) or Type 3 (29%) SMA. Patients were randomized 2:1 to receive either Evrysdi at the recommended dose or placebo. The primary efficacy endpoint was change from baseline to Month 12 in the MFM-32 score.

Important Safety Information

Avoid coadministration with drugs that are substrates of multidrug and toxin extrusion (MATE) transporters. Based on animal data, Evrysdi may cause fetal harm and may compromise male fertility. Avoid use in patients with hepatic impairment.

Please see full Prescribing Information and below for additional Important Safety Information.

Evrysdi is the first and only at-home treatment for SMA 1

Select dosing instructions 1

Please refer your patients or their caregivers to the Instructions for Use for complete information on how to take or administer Evrysdi.

Instruct patients to:

The right dose is different for each patient 1

Dosing for infants, children, and adults depends on age and weight

  • If a patient misses a planned dose and ≤6 hours have passed, he or she should take Evrysdi as soon as possible, then resume the usual dosing schedule on the next day
  • If a patient misses a planned dose and >6 hours have passed, he or she should skip the missed dose, then take the next dose at the regularly scheduled time on the next day
  • If a dose is not fully swallowed or vomiting occurs after administration, another dose should not be taken to make up for the lost dose; the next dose should be taken the following day at the regularly scheduled time
  • The liquid solution should be refrigerated (never frozen) in the original amber bottle to protect from light, and should be stored in an upright position to avoid spillage

It is recommended that you advise your patients or their caregivers about how to prepare and administer Evrysdi before the first daily dose.

*Patients (n=21) were enrolled in 1 of 2 dose cohorts. Patients in the higher-dose cohort (n=17) had their dose adjusted to the recommended dose of 0.2 mg/kg before 12 months of treatment, while patients in the low-dose cohort (n=4) did not.
Patients who died or who were withdrawn from the study are considered to be nonresponders (n=2 at Month 12). 4
Permanent ventilation 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.
§The least square (LS) mean difference for change from baseline in MFM-32 score (95% CI).
||Based on the missing-data rule for MFM-32, 6 patients (n=5 for Evrysdi; n=1 for placebo) were excluded from the analysis.
The mixed-model repeated-measures analysis included the change from baseline total score as the dependent variable; independent variables included the baseline total score, treatment group, time, treatment-by-time interaction, and the randomization stratification variable of age group (2 to 5, 6 to 11, 12 to 17, and 18 to 25 years). The MFM-32 total score was calculated according to the user manual as a percentage of the maximum score for the scale (ie, sum of the 32 item scores divided by 96 and multiplied by 100).

BSID=Bayley Scales of Infant and Toddler Development-Third Edition; MFM-32=Motor Function Measure-32 Items.

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