Onasemnogene abeparvovec intravenous (Zolgensma®) is a one-time, in vivo gene replacement therapy, for use in babies aged 12 months and under, that utilises an adeno-associated virus serotype 9 (AAV9) vector, modified to contain a functional copy of the human SMN1 gene. It is administered intravenously, allowing the vector to deliver the gene into motor neurones and peripheral tissues, enabling the sustained expression of survival motor neuron (SMN) protein, which is deficient or mutated in individuals with SMA.

Onasemnogene abeparvovec intrathecal (OAV101 IT) is a one-time in vivo gene replacement therapy for patients with SMA, proposed to treat individuals with SMA aged 2 years and over. This intrathecal injection will be used to treat a distinct cohort of SMA patients to Zolgensma. It will be assessed via NICE’s technology appraisal programme, with an expected final guidance publication date in late 2026.

Lot 1

Lot 1: Zolgensma only (if no positive NICE recommendation for OAV101 IT)

Lots 3

Lot 3: Positive NICE recommendation for OAV101 IT for adult patients (either standalone or in combination with Lots 1 and 2)

Lot 2

Lot 2: Positive recommendation for OAV101 IT for paediatric patients (in combination with Lot 1 for Zolgensma)