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    • Another delay in USD...

    Another delay in USD 2.1 million gene therapy Zolgensma data reporting, Novartis admits mistake

    Medical Dialogues BureauWritten by Medical Dialogues Bureau Published On 2019-11-03T09:45:53+05:30  |  Updated On 16 Aug 2021 2:18 PM IST

    The USFDA on Wednesday placed a partial hold on a Novartis trial of the $2.1 million-per-patient gene therapy, Zolgensma after the company informed the agency about a primate study that raised concerns of possible nerve damage or loss.


    New Delhi: Novartis scientists learnt months ago about safety concerns surrounding its gene therapy Zolgensma but delayed telling the U.S. Food and Drug Administration (FDA) due to what the Swiss drugmaker said was a "mistake".


    The FDA on Wednesday placed a partial hold on a Novartis trial of the $2.1 million-per-patient gene therapy after the company informed the agency about a primate study that raised concerns of possible nerve damage or loss.


    Novartis, under fire since August for not telling regulators about separate data manipulation allegations until after Zolgensma was approved, said scientists at its AveXis unit learnt of the primate study's findings in March.


    AveXis, which is developing Zolgensma, planned to include the findings in an annual update planned for September to the FDA but never delivered the news, Novartis said.


    "Unfortunately, a mistake was made, and this update was not implemented," Novartis said in a statement on Friday. "We notified investigators and FDA at the end of last week and were notified about the partial clinical hold earlier this week."


    The delay was first reported this week by several publications, including industry website Fierce Pharma.


    Zolgensma is already approved in the United States for children aged up to two with the deadly muscle-wasting disease spinal muscular atrophy (SMA) and is given by infusion into the young patients' veins.


    The therapy's approval in Europe and Japan has been pushed back to next year.


    Read Also: SETBACK: Novartis Zolgensma study halted by USFDA amid safety concerns


    The trial now on hold, called STRONG, includes patients aged up to five who were to receive a higher dose of the gene therapy via a spinal, or intrathecal, infusion. The study is a key part of Novartis plan to expand use in older patients, where the company hopes to compete with a rival treatment from Biogen.


    Novartis is hoping to quickly resume the STRONG trial, which has already dosed 32 patients.


    "Our discussions with FDA are ongoing and we will work diligently with FDA to identify any additional actions necessary to resume dosing," Novartis said.


    Read Also: Novartis boosts 2019 forecasts as Cosentyx, Zolgensma shine

    Clinical TrialFood and Drug AdministrationNovartisNovartis gene therapypharmapharma companypharma newsSMAspinal muscular atrophystrong trialUSFDAZolgensmaZolgensma study
    Source : Reuters

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    Medical Dialogues Bureau
    Medical Dialogues Bureau

      Medical Dialogues Bureau consists of a team of passionate medical/scientific writers, led by doctors and healthcare researchers.  Our team efforts to bring you updated and timely news about the important happenings of the medical and healthcare sector. Our editorial team can be reached at editorial@medicaldialogues.in. Check out more about our bureau/team here

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