The U.S. Food and Drug Administration (FDA) has granted AVXS-101 Orphan Drug Designation for the treatment of all types of SMA and Breakthrough Therapy Designation, as well as Fast Track Designation, for the treatment of SMA Type 1.

The European Medicines Agency (EMA) also granted AveXis access into its PRIority Medicines (PRIME) program for AVXS-101 for the treatment of SMA Type 1.

The open-label, single-arm, single-dose, multi-center trial known as STR1VE is designed to evaluate the efficacy and safety of a one-time IV infusion of AVXS-101 in patients with SMA Type 1. The co-primary efficacy outcome measures of the trial include the achievement of independent sitting for at least 30 seconds at 18 months of age; and, event-free survival at 14 months of age. Co-secondary outcome measures include the ability to thrive, and the ability to remain independent of ventilatory support at 18 months of age.

The open-label, dose-comparison, multi-center Phase 1 trial known as STRONG is designed to evaluate the safety, optimal dosing, and proof of concept for efficacy of AVXS-101 in two distinct age groups of patients with SMA Type 2, utilizing a one-time IT route of administration. The primary outcome measure for patients less than 24 months of age at the time of dosing is the achievement of the ability to stand without support for at least three seconds. The primary outcome measure for patients between 24 months and 60 months of age at the time of dosing is the achievement of change in Hammersmith Functional Motor Scale Expanded from baseline. The secondary outcome measure for both age groups is the proportion of patients that achieve the ability to walk without assistance, defined as taking at least five steps independently while displaying coordination and balance. Developmental abilities, including motor function, will also be evaluated as exploratory objectives.

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