AveXis to Initiate Screening for Remaining Patients in Pivotal Trial of AVXS-101 for SMA Type 1

AveXis (NASDAQ:AVXS), a clinical-stage gene therapy company developing treatments for patients suffering from rare and life-threatening neurological genetic diseases, today announced that, following review of safety data and early signals of efficacy from the first three patients dosed in its pivotal trial of AVXS-101 for spinal muscular atrophy (SMA) Type 1, the company, with agreement from the U.S. Food and Drug Administration, will initiate screening for the remaining patients in the trial as per the protocol.
As quoted in the press release:

“We are pleased to share that we will be initiating screening for the remaining patients to be enrolled in STR1VE as planned, following review of safety data and early signals of efficacy from the first three patients who received a one-time IV infusion of AVXS-101,” said Dr. Sukumar Nagendran, Chief Medical Officer of AveXis. “We know there is an urgent need for new treatment approaches, and we are eager to rapidly enroll the remaining patients in the pivotal trial as we further our clinical understanding of AVXS-101.”

Click here to read the full press release.