WHY SPINRAZA? / LOW DOSE REGIMEN EFFICACY
Studies in patients with SMA showed SPINRAZA® (nusinersen)
Achieved meaningful results across a range of severities and age groups–from infants to adults1-4
Backed by the longest clinical trial
program to date in infants and children.5 Supported by extensive
real-
world evidence in older children, teens, and adults2-4
Click the tabs to see the data in each population
Supportive trial: NURTURE1,7-9
Study overview: A phase 2, open-label, multicenter, multinational, single-arm trial of the Low Dose Regimen (12 mg loading doses/12 mg maintenance doses)
Study duration: Up to ~8 years, interim results below
Participants: 25 presymptomatic patients who were genetically diagnosed with SMA (two SMN2 copies, n=15; three SMN2 copies, n=10) and were aged ≤6 weeks
Primary endpoint: Time to death or respiratory intervention
Select secondary outcomes measured: 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: Generally consistent with previous safety reporting; 25/25 infants experienced any adverse event (mild [24%], moderate [52%], severe [24%]). See below for additional safety results
CHOP INTEND, Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders; HINE-2, Hammersmith Infant Neurological Exam Section 2; SMA, spinal muscular atrophy; SMN2, survival motor neuron 2; WHO, World Health Organization.
Participants taking SPINRAZA showed improvement at the initial interim analysis after treatment ≥14 months (median 25 months, range 14-34 months)1
Primary endpoint:
The time to death or respiratory intervention* could not be estimated, as there were too few events9
100% (25/25) of patients were alive without permanent ventilation1†
84% (21/25) did not require respiratory intervention9*
The majority of patients achieved the following WHO motor milestones1:
*Respiratory intervention was defined as ventilation for ≥6 hours/day continuously for ≥7 days, or tracheostomy.
†Permanent 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.
WHO, World Health Organization.
NURTURE: 5-year update (median age 4.9 years, range 3.8-5.5): all infants still alive, and most reached specified milestones consistent with normal developmental time frames10
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 intervention10§
(22/25) of those treated achieved a maximum score on the CHOP INTEND10
Majority of patients achieved major WHO motor milestones—many in time frames consistent with those without SMA7,10∥
Results may vary based on several factors, including severity of disease.
Safety profile generally consistent with previous clinical trials7,10
NURTURE results were generally consistent with safety seen in SPINRAZA pivotal studies. After a median follow-up of 4.9 years (range 3.9-5.7), 25/25 patients 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).
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
‡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.
∥WHO motor milestone windows of achievement were determined based on the WHO Multicenter Growth Reference Study windows of achievement in healthy children.
¶Caregiver-reported age at which participants first achieved WHO motor milestones confirmed by the study site at the next study visit with a “yes” or “no” response.
AE, adverse event; CHOP INTEND, Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders; SMA, spinal muscular atrophy; SMN2, survival motor neuron 2, WHO: World Health Organization.
Learn more about the mobility measures used in the SPINRAZA clinical trials.