Novartis says data confirms benefit of Zolgensma gene therapy for babies

ZURICH (Reuters) – 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.

FILE PHOTO: The company’s logo is seen at the new cell and gene therapy factory of Swiss drugmaker Novartis in Stein, Switzerland, November 28, 2019. REUTERS/Arnd Wiegmann/File Photo

The Swiss drugmaker said last month the U.S. Food and Drug Administration (FDA) had requested an additional study to examine the therapy’s efficacy in older children receiving the treatment via a spinal infusion, which could delay broader approval of the gene therapy. (This story corrects third paragraph to show the delay pertains to older patients receiving spinal infusion, not current indication)


Reporting by Silke Koltrowitz; Editing by Riham Alkousaa


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