The gene therapy product, Zolgensma® (onasemnogene abeparvovec), administered intravenously with a single injection, has just obtained conditional marketing authorisation from the European Commission for SMN1-related proximal spinal muscular atrophy (SMA). This conditional marketing authorisation relates to babies and young children weighing less than 21 kg presenting:
- a biallelic mutation on the SMN1 gene and a type 1 SMA phenotype,
- or a biallelic mutation on the SMN1 gene and up to 3 copies of the SMN2 gene.
It is based on the positive results obtained during clinical trials showing the clinical benefits with Zolgensma® in type I, type II or pre-symptomatic SMA.
With regard to France, Zolgensma® has been dispensed in the context of a cohort ATU (Temporary Authorisation for Use) since 25 May 2020. This ATU relates to patients weighing less than 21 kg “with a clinical diagnosis of type 1 SMN1-related proximal spinal muscular atrophy (SMA) or with SMA with a biallelic mutation of the SMN1 gene and up to 3 copies of the SMN2 gene” (as indicated in the protocol for therapeutic use and the collection of information accompanying it). Zolgensma® is reserved for hospital use and will be prescribed after an opinion issued by a multidisciplinary consultation meeting within the Filnemus healthcare network.
See the Zolgensma® protocol for therapeutic use (PTU) and collection of information (in French, PDF)