- The FDA has signed off on a pivotal clinical trial, STRIVE, evaluating AveXis' (NASDAQ:AVXS) lead gene therapy candidate AVXS-101 in patients with spinal muscular atrophy (SMA) Type 1 using the intravenous (IV) formulation produced by the company's GMP commercial manufacturing process. The trial will be initiated immediately.
- GMP-caliber product will be used for all subsequent studies and commercial supply, if approved.
- STRIVE's co-primary endpoints are the ability to independently sit for at least 30 seconds at 18 months of age and event-free survival at 14 months of age.
- The company says its discussions with the FDA on the details for intrathecal (within the spine) administration of AVXS-101 in an SMA Type 2 study are continuing . It will update investors in Q4.
- Shares are up 2% premarket but only on 102 shares.
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