AveXis submits regulatory applications for AVXS-101 in the U.S., Europe and Japan for use in infants with SMA Type 1.
AveXis announced yesterday that it has submitted regulatory applications for AVXS-101 in the U.S., Europe and Japan for use in infants with SMA Type 1.
Please read their community update
Dear SMA Community,
AveXis, a Novartis company, is pleased to let you know that we have submitted regulatory applications for AVXS-101 in the U.S., Europe and Japan for use in infants with SMA Type 1. This is an important and exciting initial step toward the potential approval of AVXS-101, a gene replacement therapy candidate for the treatment of the underlying root cause of SMA. Data from our pivotal Phase 1 study conducted in SMA Type 1 formed the primary basis for these submissions. Based on the data included in the applications, our expectation is that the initial label will be for intravenous (IV) use of AVXS-101 for infants with SMA Type 1, as IV dosing has only been studied in clinical trials in infants. Our clinical development program is designed to help us understand the safety and how well AVXS-101 works in a broad set of pediatric patients with SMA. For example, our study of AVXS-101 in SMA Type 2 (STRONG) is ongoing, and data from that study will help determine the final study design for the planned study in children up to 18 years of age (REACH). Please see the table below from more information and updates on our planned and ongoing studies.
Next steps for AVXS-101 regulatory applications:
- U.S. – The U.S. Food and Drug Administration (FDA) will make the decision of whether to accept our Biologics License Application (BLA) for AVXS-101 within the next 60 days. If accepted, we anticipate the FDA will take six months to review the BLA and make a decision on whether or not to approve AVXS-101. This enables a potential approval for AVXS-101 in the U.S. in the first half of 2019. We will provide an update on anticipated approval timing if and when the BLA has been accepted for review.
- Europe – Similarly, the European Medicines Agency is evaluating our Marketing Authorization Application (MAA) for AVXS-101 and will determine its acceptability within the next couple of weeks. If accepted, the European Medicines Agency will have seven months to review the MAA and make a decision on whether or not to approve AVXS-101. This enables a potential approval for AVXS-101 in Europe in mid-2019.
- Japan – In mid-September we initiated the pre-application review period of the Japanese New Drug Application (J-NDA) for AVXS-101 with the Ministry of Health, Labour and Welfare (MHLW). Upon completion of the pre-review, we anticipate the application submission of the J-NDA to MHLW will be completed by the end of this year and will have a six-month review period once accepted. This enables a potential approval for AVXS-101 in Japan in the first half of 2019.
AveXis is excited by this progress, and we want to thank everyone in the community for your ongoing interest in AVXS-101. We especially want to thank the patients and families who have participated in our clinical trials – it is because of you that we are where we are today, and we are incredibly grateful. We look forward to continuing to keep you updated. In the meantime, if you have any questions please contact us.
The AveXis Team
- Please read the original community update. It contains answers to frequently asked questions and an overview of AVXS-101’s Clinical Development Program (as of October 2018)