Zolgensma®, initially developed by AveXis (subsequently acquired by Novartis) has just received a positive opinion in SMA by the by the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA). The CHMP recommends a conditional marketing authorization which should only concern “babies and young children”:
- suffering from proximal spinal muscular atrophy (SMA) 5q with a biallelic mutation in the SMN1 gene and a clinical diagnosis of SMA Type 1,
- or suffering from SMA 5q with a bi-allelic mutation in the SMN1 gene and up to 3 copies of the SMN2 gene.
The precise indication will be based on the decisions of each competent authority and the final decision of the European Commission of the EMA is expected to be made by June 2020.
EMA press release (03/27/2020) « New gene therapy to treat spinal muscular atrophy »
