Studies in patients with spinal muscular
atrophy (SMA) show SPINRAZA
Achieved meaningful results across
age
groups—from infants to adults1-3
Backed by the longest clinical trial
program to date in infants and
children.4 Supported by extensive
real-world evidence in
adults, teens,
and older children2,3,5-7
Click the tabs to see the data in the selected population
SPINRAZA IN NEWBORNS/INFANTS
NURTURE
The first and longest trial in presymptomatic SMA with proven efficacy1,4
Supportive trial: NURTURE1,8-10
Study: A phase 2, open-label, multicenter, multinational, single-arm trial
Treatment duration: Ongoing; SPINRAZA (12 mg)
Participants: 25 presymptomatic infants who were genetically diagnosed with SMA (2 SMN2 copies, n=15; 3 SMN2 copies, n=10) and were aged ≤6 weeks
Primary endpoint: Time to death or respiratory intervention
Secondary endpoints: Attainment of WHO motor milestones and change from baseline in the CHOP INTEND motor function scale
Study limitations: Small participant group size and no sham control group; CHOP INTEND score was assessed only until a participant achieved the maximum score of 64. HINE-2 score was assessed until day 778, but not at later study visits
Safety: Consistent with the SPINRAZA Prescribing Information; after a follow-up of 14 months to 5.7 years, no new safety concerns were identified and 100% (25/25) of participants had an adverse event that was mild, or moderate in severity.
CHOP INTEND, Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders; HINE-2, Hammersmith Infant Neurological Exam Part 2; SMA, spinal muscular atrophy; SMN2, survival motor neuron 2 gene; WHO, World Health Organization.
Participants taking SPINRAZA showed improvement at the initial interim analysis at 14 months (median 25 months, range 14-34 months)1
Primary endpoint:
100% (25/25) of infants were alive without permanent ventilation1*
84% (21/25) did not require respiratory intervention8†
The majority of infants achieved the following WHO motor milestones1:
*Permanent assisted ventilation was defined as tracheostomy or ventilator support for ≥16 hours per day for >21 continuous days in the absence of an acute reversible event.1,8
†Respiratory intervention was defined as ventilation for ≥6 hours/day continuously for ≥7 days, or tracheostomy.
WHO, World Health Organization.
NURTURE: 5-year update (median age 4.9 years, range 3.8-5.5): all infants still alive, and most patients reached specified milestones consistent with normal development time frames8
After nearly 5 years of being treated with SPINRAZA,
(25/25) of children were alive and did not require permanent ventilation*; 16% (4/25) required respiratory intervention8†
*Defined as: ventilation for ≥16 hours/day continuously for >21 days in the absence of an acute reversible event, or tracheostomy.
†Defined as: ventilation for ≥6 hours/day continuously for ≥7 days, or tracheostomy.
(22/25) of those treated achieved a maximum score on the CHOP INTEND8
Majority of infants achieved major WHO motor milestones (MM)—many in time frames consistent with those without SMA8‡
Results may vary based on several factors, including severity of disease.
‡WHO motor milestone windows of achievement were determined based on the WHO Multicenter Growth Reference Study windows of achievement in healthy children.
Well-established safety generally consistent with previous clinical trials8
NURTURE results were consistent with safety seen in SPINRAZA pivotal studies. After a median follow-up of 4.9 years (range 3.9-5.7), 25/25 infants experienced an adverse event (any, including mild [24%], moderate [52%], severe [24%]).§
§Serious AEs (N=12) were tendon disorder, dehydration (n=1); bronchitis, choking, pneumonia, medical device change, tonsillectomy (n=1); pneumonia, tonsillectomy (n=1); upper respiratory tract infection, diarrhea (n=1); mycoplasmal pneumonia (n=1); viral upper respiratory tract infection (n=1); post lumbar puncture syndrome, abdominal distention, respiratory distress, dehydration, enterovirus infection, corona virus infection (confirmed not COVID-19), respiratory syncytial virus bronchiolitis, bacterial pneumonia, acute respiratory failure, respiratory failure, tachycardia, viral gastroenteritis, pneumonia, feeding disorder, choking (n=1); respiratory distress, respiratory syncytial virus bronchiolitis, pneumonia aspiration, pneumonia, influenza A virus positive, respiratory failure, respirovirus positive test (n=1); failure to thrive (n=1); urinary tract infection (n=1); respiratory syncytial virus infection (n=1); pyrexia, pneumococcal pneumonia, pneumonia, pseudomonal pneumonia, upper respiratory tract infection (n=1).
CHOP INTEND, Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders; MM, motor milestone; SMA, spinal muscular atrophy; SMN2, survival motor neuron 2 gene; WHO, World Health Organization.
NURTURE study interim analysis data cut-off date: February 15, 2021.
- One additional child achieved walking alone between the February 19, 2020 and February 15, 2021 data cuts||
- There was no loss of major motor milestones
||Caregiver-reported age at which participant first achieved WHO motor milestones confirmed by the study site at the next study visit with a “yes” or “no” response.
Learn more about the mobility measures used in the SPINRAZA clinical trials.
