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Novartis Issues Update On AVXS-101 Intrathecal Clinical Development Program

Novartis International AG (NVS) announced that Novartis Gene Therapies recently received feedback from the US Food and Drug Administration or FDA following their review of data from the STRONG study of the intrathecal or IT formulation of AVXS-101 in older patients with spinal muscular atrophy or SMA.

The FDA has acknowledged the potential of AVXS-101 IT in this patient population and recommends a pivotal confirmatory study to supplement the existing STRONG data and further support the regulatory submission for AVXS-101 IT.

This guidance provides clarity on the path to registration for AVXS-101 IT. The company noted that the trial design and other details are being evaluated and a comprehensive update on the overall Novartis SMA clinical development program would be provided at a future time following further discussions with health authorities.

This request for a study is unrelated to the partial clinical hold on AVXS-101 IT, and the new study would not be initiated in the US until the hold has been lifted by the FDA. Novartis Gene Therapies remains confident in the overall benefit-risk profile for patients on treatment. This does not impact marketed Zolgensma and the company continues to advance its regulatory filings and intravenous clinical studies.

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