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British baby’s death not caused by Zolgensma says Novartis

Published on 19/09/19 at 10:19am

Swiss drugmaker Novartis have stated that a British baby’s death this year after getting Novartis’ gene therapy Zolgensma, was not caused by a toxic drug reaction allaying concerns over the $2.1 million-per-patient treatment’s risks.

Novartis also stated that babies with muscle-wasting spinal muscular atrophy (SMA) treated before symptoms emerge were meeting normal development milestones, such as crawling and walking, an announcement that has been welcomed in lieu of the recent data manipulation scandal that has shrouded Zolgensma.

Novartis has been investigating the death of a 6 month year-old patient in a European trial with investigators and coroner concluding the immediate cause of death was brain damage from oxygen deprivation after respiratory stress.

While gene therapy may have contributed to hospitalisation of the baby due to abnormal liver function and low blood pressure, these side effects have been known about since before its approval.

AveXis Chief Executive Dave Lennon, said: “We had been concerned about the potential… that Zolgensma might have been causing brain inflammation.

“In this case, the original diagnosis, and that association hasn’t held true.”

Zolgensma is the world’s most expensive one-time medical treatment and has provided hope for families facing a devastating diagnosis of SMA, the leading genetic cause of infant death.

After the FDA began probing manipulation of early data by AveXis scientists, saying it could results in criminal or civil action, Novartis replaced AveXis employees and since promised to speed disclosures of problematic data; with the FDA leaving it on the market concluding it is safe and effective.

Today’s news comes just as AveXis announced new interim data from the Phase 3 SPR1NT trial which demonstrated significant efficacy with children aged over 4 years continuing to achieve developmental milestones.

Eugenio Mercuri of the Department of Paediatric Neurology, Catholic University, Rome, said: “These updated reinforce what we have seen in other Zolgensma studies, including survival of children with SMA type 1 who would have in the past died or required permanent ventilation before the age of two.

“We are seeing further robust evidence of the potential of gene therapy to effectively halt motor neuron loss, help patients achieve motor milestones and alter the course of SMA with a one-time treatment.”

Nik Kiran

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