AveXis to Use Intended Commercial GMP Product in SMA Type 2 Study
AveXis, Inc., a clinical-stage gene therapy company developing treatments for patients suffering from rare and life-threatening neurological genetic diseases, today reported that, based on emerging data from its intended commercial Good Manufacturing Practice (GMP) development work and ongoing discussions with the U.S. Food and Drug Administration (FDA), the company has made the strategic decision to use the intended commercial GMP derived product in all future studies of the company’s proprietary gene therapy candidate, AVXS-101, including the planned spinal muscular atrophy (SMA) Type 2 trial.
The company had originally planned to use existing inventory for its initial trial in SMA Type 2, and to then use its intended commercial GMP derived product in later Type 2 trials. By using the intended commercial GMP derived product for the duration of its study of AVXS-101 in Type 2 patients, the need for a comparability analysis later in program development is eliminated, potentially accelerating the timeframe for gathering clinical data that may be used as part of a data set to support a future SMA Type 2 indication.
"We believe this strategic decision has the potential to streamline the development of the clinical data set for AVXS-‐101 in SMA Type 2," said Sean Nolan, President and Chief Executive Officer of AveXis. "We are pleased with the advances we have seen from our process development efforts and believe the optimal approach is to use the scalable process in our clinical evaluation of AVXS-‐101 in SMA Type 2 from the outset."
The company now expects to initiate the planned study of AVXS-101 in SMA Type 2 patients in Q2 2017, assuming a positive outcome from its Type B manufacturing meeting with the FDA, currently anticipated to occur in Q1 2017.
This approach is intended to potentially expedite the SMA Type 2 program and does not impact the development program for AVXS-101 in SMA Type 1. AveXis expects to initiate a U.S. pivotal trial of AVXS-101 in patients with SMA Type 1 in the first half of 2017, and plans to use the same intended commercial GMP derived product for that study.
For More Information
To learn more about the gene therapy program and upcoming clinical trials, please see a recent announcement from our news blog.