Novartis Issues Community Statement with Update on AVXS-101 Intrathecal Clinical Development Program

Dear SMA Community,

We recently received important feedback from the U.S. Food and Drug Administration (FDA) following their review of data from the STRONG study of AVXS-101 intrathecal (IT) formulation in older patients with spinal muscular atrophy (SMA) and we’d like to share an update with you.  (Please see the press release issue by Novartis on 9/23/20.)

The FDA has acknowledged the potential of AVXS-101 in this patient population, but recommended a pivotal confirmatory study to supplement existing data, and further support filing for AVXS-101 IT. Given we will need to initiate a pivotal confirmatory trial for the IT formulation, we will no longer be submitting a filing for the IT formulation in 2021 as previously targeted. The public statement with additional details is available on  It’s important to note that 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 U.S. until the hold has been lifted by the FDA.

As Novartis Gene Therapies, we remain unwavering in our commitment to the SMA community and this guidance is very helpful in providing clarity on the best path toward registration of our IT formulation. It is important to reinforce this does not impact marketed Zolgensma® (omnasemnogene abeparvovec-xioi) and we continue to advance our global regulatory filings and intravenous clinical studies. More than 600 children have been treated with Zolgensma to date, and we expect this number will continue to grow as we pursue additional approvals worldwide. The pioneering initiatives we are pursuing together, stand to deliver lasting impact for our SMA community. We will continue to advance our work on behalf of patients, with focus and determination. Our goals in the SMA community are ambitious, and we can only achieve them by working together. We are grateful for your continued commitment and look forward to collaborating on new programs that provide even more promise for more patients. Thank you for your continued partnership to help create a lifetime of possibilities.


Your Novartis Gene Therapies Advocacy Team


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