Novartis says data confirms benefit of Zolgensma gene therapy for babies

Novartis said new interim data from an ongoing phase clinical trial for its $2.1 million-per-patient gene therapy Zolgensma showed spinal muscular atrophy patients experienced significant therapeutic benefit.

Nearly two-thirds of patients aged less than six months in the STR1VE-EU study have already achieved developmental motor milestones not observed in natural history of SMA type 1, a rare genetic disease, at a mean duration of follow-up of 10.6 months, Novartis said in a statement on Thursday.

The Swiss drugmaker said last month that Zolgensma faced a possible delay after the U.S. Food and Drug Administration (FDA) requested an additional study to examine the therapy’s efficacy in older children.

https://www.reuters.com/article/us-novartis-zolgensma/novartis-says-data-confirms-benefit-of-zolgensma-gene-therapy-for-babies-idUSKBN26M4N0