AveXis Releases Community Statement on STR1VE

By Cure SMA | Published On February 1, 2018

AveXis Releases Community Statement on STR1VE

AveXis has provided the following community statement on AVXS-101.

Dear SMA Community,

AveXis, the gene therapy company developing a new approach to treat SMA known as AVXS‐101, is pleased to initiate screening for the remaining patients to be enrolled in the pivotal trial of AVXS-101 for SMA Type 1, known as STR1VE.

This next step in the STR1VE study comes after a review of the information gathered from the first three patients who were dosed in the trial. There was at least a four-week interval scheduled between dosing the first three patients to allow review of the safety analysis as well as early signals of efficacy.

STR1VE is designed to evaluate the efficacy and safety of a one-time intravenous (IV) infusion of AVXS-101.

  • OVERVIEW: STR1VE is an ongoing study of AVXS-101 in patients with SMA Type 1, studied at multiple centers across the U.S. which began enrolling in September 2017.
  • ADMINISTRATION: In STR1VE, AVXS-101 is given one-time through a needle inserted into a vein, known as an intravenous (IV) infusion.
  • WHO: STR1VE will enroll a minimum of 15 patients with SMA Type 1 who are less than six months of age at the time of gene therapy administration, and who have one or two copies of the SMN2 backup gene and bi-allelic SMN1 gene deletion or point mutations.

We know there is an urgent need for new treatment approaches, and we are eager to rapidly enroll the remaining patients in the pivotal trial as we further our clinical understanding of AVXS-101.

If you have any questions about STR1VE, please contact us at medinfo@avexis.com.


The AveXis Team


1. When will AVXS-101 be approved by the FDA? 

  • We cannot speak on behalf of the FDA regarding whether and when they will decide to approve AVXS-101. 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 what type(s) of patients.

2. Who should I contact to see if my child is eligible for STR1VE? 

3. I saw reports that gene therapy may not be safe. Does that apply to AVXS-101? 

  • We understand that a recently published paper discussing a small, early-stage study of AAVHu68 in animals may have raised some concerns. It is important to know that this does not have implications for AVXS-101, primarily because AAVHu68 is a different vector than AAV9 – the adeno-associated virus in AVXS-101. Therefore, direct comparisons of the two cannot be made.

Cure SMA Webinar on Treatment Access and Clinical Trials 

Be sure to register for our webinar on Thursday, February 15th at 12:00pm CT to learn more about ongoing and upcoming clinical trials, including the AVXS-101 program. 

We look forward to hosting an open, candid, and informal discussion of where our community stands on issues surrounding treatment access, and on the status of clinical trials.


Topics: Clinical Trials, Research, Front Page News

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