FIREFISH is a 2-part, open-label trial in infantile-onset spinal muscular atrophy (SMA) studying the safety and efficacy of Evrysdi in 62 infants 2 to 7 months old with Type 1 SMA. Part 1 determined the recommended dose (N=21). Part 2 assessed the efficacy and safety of Evrysdi (N=41). A pooled analysis of 58 infants aged 2 to 7 months received the recommended dose of Evrysdi in Parts 1 and 2. Infants reflected those seen in a real-world setting, including age, disease progression, baseline motor function, and levels of disease severity.1,14
SITTING ABILITY
as measured by BSID-III, Item 22
Recommended dose
AFTER 12 MONTHS OF TREATMENT
AFTER 12 MONTHS OF TREATMENT
AFTER 24 MONTHS OF TREATMENT
BSID-III=Bayley Scales of Infant and Toddler Development–Third Edition.
MOTOR MILESTONES AFTER 24 MONTHS OF TREATMENT
Recommended-dose cohort
BSID-III=Bayley Scales of Infant and Toddler Development–Third Edition; 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.
All-patients cohort (N=62)*
AFTER 12 MONTHS OF TREATMENT
of infants (54/62) were alive without permanent ventilation†
AFTER 24 MONTHS OF TREATMENT
of infants (52/62) were alive without permanent ventilation†
*All-patients cohort, dose adjusted per protocol. Intent-to-treat population.
†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.
84% of infants were estimated event-free after 24 months of treatment2*†
All-patients cohort (N=62)
*Defined as alive with no permanent ventilation.
Permanent ventilation is 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.
†Six infants died (4 within the first 3 months following study enrollment) and one additional patient withdrew from treatment and died 3.5 months later. Four patients required permanent ventilation by month 24.
‡Two patients were censored because they attended the month 24 visit early, one patient was censored after discontinuing treatment and died 3.5 months later.
As of clinical cut-off date: November 12, 2020.
AFTER 24 MONTHS OF TREATMENT11
As of clinical cut-off date: November 12, 2020.
*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. Clinical cut-off date for the 12-month data: November 14, 2019; clinical cut-off date for 24-month data: November 12, 2020.
†Data are available for only 52 infants in FIREFISH Parts 1 (Cohort B, n=17) and 2 (n=41) because of 6 infant deaths prior to the time of the assessment. One infant died approximately 3.5 months after discontinuing treatment.
‡Includes infants who were able to feed orally or in combination with a feeding tube.
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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Darras BT, Boespflug-Tanguy O, Day JW, et al, on behalf of the FIREFISH Working Group. FIREFISH Parts 1 and 2: 24-month safety and efficacy of risdiplam in infants with Type 1 SMA. Poster presented at: Muscular Dystrophy Association Clinical and Scientific Conference; March 13-16, 2022; Nashville, TN.
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