Teaser case. Selected price and peak-sales fields are held for a paid Brief — the rest is the method.
Algorithm
NBS → urgent GT if eligible and seronegative; oral if not.
Evrysdi default for convenience; Spinraza where IT preferred or GT already given.
Older / previously untreated
Itvisma or chronic SMN. Case-by-case.
One-time, early / Type 1
Zolgensma
onasemnogene abeparvovec · Novartis
List / typical annual
$2.1M
One-time ~$2.1M
- Eligible
- Bi-allelic SMN1, age/weight per label
- Route
- One-time IV
- Endpoint that matters
- Survival / permanent ventilation; CHOP-INTEND
- Curative?
- Potentially transformative
EfficacyTransforms Type 1 natural history when given early. Event-free survival and motor milestones vs historical.SafetyAcute liver injury, troponin, thrombocytopenia. AAV9 serostatus. Age/weight label limits.Residual unmet needCannot redose. Older / heavier patients historically out of label — Itvisma is the answer.