Novartis provides update on AVXS-101 Intrathecal Clinical Development Program for spinal muscular atrophy.

Novartis Gene Therapies recently received feedback from the FDA following their 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. Novartis Gene Therapies remains confident in the overall benefit-risk profile for patients on treatment. This does not impact marketed Zolgensma (onasemnogene abeparvovec) and the company continues to advance its regulatory filings and intravenous clinical studies. Novartis Gene Therapies reaffirms its commitment to the SMA community and to pursuing solutions for patients with all types of SMA, including older children and adults. All patients deserve a gene therapy designed to address the genetic root cause of their disease with a single dose. Zolgensma is approved in the US, Japan and, most recently, Brazil, for patients with SMA under the age of two. Zolgensma also continues to have a strong launch in Europe where it is approved for babies and young children with a clinical diagnosis of SMA Type 1 or SMA with up to three copies of the SMN2 gene, with dosing guidance provided up to 21 kg. More than 600 patients have benefited from Zolgensma, including through clinical trials, commercially and through the managed access program. This number is expected to continue to grow as this transformative gene therapy is approved in additional markets and as the company pursues additional studies to fully explore the impact of Zolgensma across a broad population of patients with SM.