AveXis to Imminently Initiate AVXS-101 Pivotal Trial in the US

12 October 2017

The Food and Drug Administration (FDA) has granted permission to AveXis to begin their planned US pivotal trial of their gene therapy drug for SMA, AVXS-101. Avexis say that they are to immediately begin the trial.

AVXS-101, which has “Breakthrough Therapy” status to facilitate the drug approval process (for more information click here), uses harmless, genetically engineered viruses to increase SMN protein levels. Recently reported results from a Phase I trial, indicate that the therapy is safe and well tolerated by young SMA patients (for more information click here).

This new pivotal study, which has been called STR1VE and will include at least 15 patients with SMA type 1, will have an open-label, single-arm design, meaning that everyone will receive the drug, and no placebo will be used. As the trial is designed to continue to assess safety, but also to evaluate drug effectiveness, motor milestone achievements of patients on the drug will be compared to natural history information from untreated SMA patients.

More specifically, the trial will determine if AVXS-101 improves the ability of patients to sit unaided for at least 30 seconds, and whether it increases the time from birth to a disease-associated event. An event is defined as the time when ventilation support for breathing is required for at least 16 hours a day for 14 consecutive days, or sadly when a patient dies.

The drug will be produced using Good Manufacturing Practice (click here for more information) and will be given as a single injection into the blood at a dose equivalent to the higher concentration administered to Cohort 2 in the Phase I study.

A clinical trial with SMA type 2 patients and a pivotal trial in Europe are both planned for AVXS-101 in the near future (click here for further information). 


Further Information

AveXis Press Release