In March, Novartis was able to announce that Zolegensma (onasemnogene abeparvovec) that it had received a positive opinion on its application for marketing approval by the European Medicines Agency.
Yesterday this was followed up by the announcement that the European Commission (EC) had provided the gene therapy conditional approval for the treatment of patients with spinal muscular atrophy. The treatment will be available for those with a bi-allelic mutation in the SMN1 gene and a diagnosis of SMA type 1; or for patients with SMA with the mutation and up to three copies of the SMN2 gene.
The company added that the approval also covers babies and young children with SMA, up to a body weight of 21kg (46 pounds).
Novartis stated that the treatment will be made available immediately in France and that Germany would gain access ‘shortly’.
In terms of pricing, the company noted that it was working with stakeholder organizations across Europe, so that pricing would "work within existing, local pricing and reimbursement frameworks."
The pricing has proved a controversial aspect of the one-time gene therapy, which entered the US market at a cost of $2.1m (€1.9m) per patient.
In its announcement, the company countered that the cost of caring for a child with SMA to the healthcare system is approximately €2.5m to €4m for 10 years of care.
In fourth quarter results, the treatment saw sales of $170m in the US.