In FIREFISH Part 1, efficacy for infantile-onset SMA was established
based on the ability to sit without support and survival without
permanent ventilation
FIREFISH is an open-label, 2-part study to investigate the efficacy,
safety, and tolerability of Evrysdi in infants with Type 1 SMA. Learn
more about the FIREFISH trial design.
Sitting milestone
Evrysdi helped infants achieve an important motor milestone
Infants achieved the ability to sit without support for at least 5
seconds as measured by BSID-III 1
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 additional important Safety Information below and full
Prescribing Information.
*Patients (n=21) were enrolled in one of two 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†Patients who died or who were withdrawn from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).
8
BSID-III=Bayley Scales of Infant and Toddler
Development–Third Edition; CHOP INTEND=Children’s Hospital of
Philadelphia Infant Test of Neurological Disorders;
HINE-2=Hammersmith Infant Neurological Examination Module 2.
Survival data
Evrysdi helped infants survive without permanent ventilation
Results after at least 12 months of treatment demonstrated efficacy during a critical time for infants with SMA 1
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 additional important Safety Information below and full
Prescribing Information.
*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.
BSID-III=Bayley Scales of Infant and
Toddler Development-Third Edition; CHOP INTEND=Children’s Hospital
of Philadelphia Infant Test of Neurological Disorders;
HINE-2=Hammersmith Infant Neurological Examination Module 2.
Additional data
Exploratory endpoints for feeding and swallowing ability 8*
After 12 months of treatment in the recommended-dose cohort (n=17)†:
- 88% (15/17) were able to feed orally
- 88% (15/17) were able to swallow
Feeding and swallowing assessments should be interpreted with caution, as the natural history of these outcomes is not well characterized in the infantile-onset population.
Motor milestone data in FIREFISH Part 1 from assessments using HINE-2 8
HINE-2 data should be interpreted with caution, as the natural history of these motor function endpoints is not well characterized in the infantile-onset population.
77% (13/17) of infants in the recommended-dose cohort met prespecified criteria for developmental milestones 8
- Infants who met
prespecified criteria showed an improvement in more motor milestones
than showed worsening‡
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 additional important Safety Information below and full
Prescribing Information.
Motor function data in FIREFISH Part 1 from assessments using
CHOP INTEND
CHOP INTEND data should be interpreted with caution, as the natural history of these motor function endpoints is not well characterized in the infantile-onset population.
- At baseline, the mean CHOP INTEND score for the recommended-dose cohort was 24.3 (16.0-34.0)
*Assessed using nutritional status interview of
caregiver and a standard swallowing assessment based on local
practice and performed by a qualified individual. Swallowing
assessment completed at baseline and during follow-up visits.
Ability of the patient to swallow age appropriate foods was
assessed.
†Ability to feed orally includes
patients exclusively fed orally (n=13) and in combination with a
feeding tube (n=2). One patient could not swallow at baseline.
‡An improvement in a motor milestone is defined
as at least a 2-point increase in ability to kick or a 1-point
increase in head control, rolling, sitting, crawling, standing, or
walking. A worsening in motor milestone is defined as a 2-point
decrease in ability to kick or a 1-point decrease in head control,
rolling, sitting, crawling, standing, or walking. Excludes voluntary
grasp.
BSID-III=Bayley Scales of Infant and Toddler
Development-Third Edition; CHOP INTEND=Children’s Hospital of
Philadelphia Infant Test of Neurological Disorders;
HINE-2=Hammersmith Infant Neurological Examination Module 2.