Novartis progresses AVXS-101 IT development program

Novartis Gene Therapies, the new name for AveXis - which Swiss pharm giant Novartis (NOVN: VX) acquired in an $8.7 billion deal in 2018, has recently received feedback from the US Food and Drug Administration following its review of data from the STRONG study of the intrathecal (IT) formulation of AVXS-101 in older patients with spinal muscular atrophy (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. Trial design and other details are being evaluated and a comprehensive update on the overall Novartis SMA clinical development program will 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 will not be initiated in the US until the hold has been lifted by the FDA.