AveXis Issues Community Statement on the SPR1NT Trial

By Cure SMA | Published On May 8, 2018

AveXis Issues Community Statement on the SPR1NT Trial

AveXis has provided the following community statement on the SPR1NT trial.

Dear SMA Community,

AveXis, the gene therapy company developing a new approach to treat SMA known as AVXS‐101, recently treated the first patient in a new study known as SPR1NT. SPR1NT is a multi-national study that will evaluate AVXS-101 in approximately 44 patients less than 6 weeks old with SMA Types 1, 2 or 3 who have 2, 3 or 4 copies of SMN2 – often called the SMA back-up gene – and have not yet shown symptoms of the disease, also referred to as being “pre-symptomatic.”

As with all of our studies, the intention of SPR1NT is to further our understanding of both the safety of AVXS-101, and how well AVXS-101 may work in SMA patients. This study is specifically to look at patients who are pre-symptomatic and less than 6 weeks old. We are excited by the progress we are making, bringing us ever closer to our goal of making AVXS-101 available to patients and their families.

For more information regarding the study, please read our press release here or view the listing on ClinicalTrials.gov. If you have any questions about SPR1NT, please contact us at medinfo@avexis.com.

The AveXis Team


  • OVERVIEW: SPR1NT is a multi-national trial in pre-symptomatic patients with SMA Types 1, 2 and 3 who have 2, 3 or 4, copies of SMN2 and are less than 6 weeks old.
  • ADMINISTRATION: In SPR1NT, AVXS-101 is administered through a one-time intravenous (also known as IV) infusion.
  • WHO: SPR1NT will enroll approximately 44 patients with two, three or four copies of SMN2 who are less than six weeks of age and pre-symptomatic at the time of gene therapy administration.


  1. When will AVXS-101 be approved by the FDA?
    • While we cannot speak on behalf of the FDA regarding whether they will decide to approve AVXS-101 and when, the goal of the ongoing and planned studies is to enhance our understanding of how well AVXS-101 works and, more importantly, its safety profile in a broad range of people with SMA. The FDA then uses the information collected in these clinical studies to help determine whether AVXS-101 should be approved, and, if so, for use in what patients.
  2. Who should I contact to see if my child is eligible for SPR1NT?

Topics: Clinical Trials, Front Page News

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