AveXis Gets FDA Breakthrough Therapy Status for Its AVXS-101 Gene Replacement Therapy

AveXis, Inc.,

disclosed that the Food and Drug Administration (FDA) has granted Breakthrough Therapy status for its AVXS-101, its lead development candidate for the treatment of spinal muscular atrophy (SMA) Type 1 in pediatric patients. AveXis said that the Breakthrough Therapy Status was based on preliminary clinical results from the ongoing sstudy of AVXS-101, which was conducted in collaboration with The Research Institute at Nationwide Children's Hospital and The Ohio State University. The company revealed that the regulator has requested it to submit a Type B meeting request for a multidisciplinary, comprehensive discussion of the development program for AVXS-101. AveXis added that it intends to submit the meeting request later this month. Its president and CEO, Sean Nolan, commented, "We are encouraged to have received Breakthrough Therapy Designation for AVXS-101, and look forward to collaborating with the FDA to determine next steps in the development pathway for AVXS-101. By this action the FDA recognizes the high unmet need for effective treatment options for patients suffering from SMA." Following the news, the stock traded 8.30 percent higher at the time of writing this on Wednesday.