AveXis gets nod from FDA to start pivotal SMA trial

AveXis Inc. (NASDAQ:AVXS) gained $4.54 to $96.73 on Friday after the company announced plans to "immediately" start the pivotal STR1VE trial of AVXS-101 to treat spinal muscular atrophy. AVXS-101 is a gene therapy that delivers the survival motor neuron (SMN) gene using NAV rAAV9 vector technology from RegenxBio Inc. (NASDAQ:RGNX).

AveXis said FDA notified the company it could initiate the trial following a review of information regarding the company's proposed potency assay and GMP manufacturing process for AVXS-101. AveXis said its GMP manufacturing process incorporates an adherent cell culture approach that differs from the Hyperstacks approach used by Nationwide Children's Hospital to manufacture the product for a Phase I trial of AVXS-101 in SMA type I patients...