Tag: avxs-101

News, Research

AveXis Releases Letter to SMA Community: FDA Places Partial Hold on Clinical Trials for Intrathecally Delivered AVXS-101

MDA Staff

On Oct. 30, AveXis released a letter to the spinal muscular atrophy (SMA) community regarding a decision by the US Food and Drug Administration (FDA) to place a partial hold on clinical trials for intrathecal (IT) administration of AVXS-101. The full letter follows. The order stops enrollment of patients in the high-dose group of AveXis’ . . .

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News, Research

FDA Accepts AveXis’ BLA for SMA Drug Zolgensma Under Priority Review

MDA Staff

Today, Novartis, the parent company of AveXis Inc., announced that the U.S. Food and Drug Administration (FDA) has accepted the company’s Biologics License Application (BLA) under Priority Review for Zolgensma (formerly known as AVXS-101), a gene therapy for the treatment of spinal muscular atrophy (SMA) type 1. Priority Review status requires the FDA to review . . .

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