Novartis Provides Update on AVXS-101 Intrathecal Clinical Development Program

  • Novartis Gene Therapies to initiate new pivotal confirmatory study to evaluate use of AVXS-101 intrathecal (IT) formulation in older patients with SMA to further support registration

Basel, September 23, 2020 - Novartis Gene Therapies recently received feedback from the US Food and Drug Administration (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 SMA.

The company’s commitment to SMA extends beyond gene therapy to branaplam (LMI070), an oral, once-weekly RNA splicing modulator also currently under development, to expand the treatment options for SMA patients. 

About Zolgensma (onasemnogene abeparvovec)
Zolgensma is designed to address the genetic root cause of SMA by providing a functional copy of the human SMN gene to halt disease progression through sustained SMN protein expression with a single, one-time IV infusion. Zolgensma was approved by the US Food and Drug Administration in May 2019 and represents the first approved therapeutic in the company’s proprietary platform to treat rare, monogenic diseases using gene therapy. In addition to the US, Zolgensma is approved in Japan, Europe and Brazil. More than 600 patients have been treated with Zolgensma, including clinical trials, commercially and through the managed access program. Novartis Gene Therapies is pursuing registration in close to three dozen countries with regulatory decisions anticipated in Switzerland, Canada, Israel, Australia, and South Korea in late-2020 or early 2021.

About Spinal Muscular Atrophy
SMA is the leading genetic cause of infant death. If left untreated, SMA Type 1 leads to death or the need for permanent ventilation by the age of two in more than 90% of cases.
SMA is a rare, genetic neuromuscular disease caused by a lack of a functional SMN1 gene, resulting in the rapid and irreversible loss of motor neurons, affecting muscle functions, including breathing, swallowing and basic movement. It is imperative to diagnose SMA and begin treatment, including proactive supportive care, as early as possible to halt irreversible motor neuron loss and disease progression. This is especially critical in SMA Type 1, where motor neuron degeneration starts before birth and escalates quickly. Loss of motor neurons cannot be reversed, so SMA patients with symptoms at the time of treatment will likely require some supportive respiratory, nutritional and/or musculoskeletal care to maximize functional abilities. More than 30% of patients with SMA Type 2 will die by age 25.

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