AveXis’s gene therapy candidate receives Breakthrough Therapy Designation from FDA

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AveXis, a clinical-stage gene therapy company specialized in the development of treatments for orphan and life-threatening neurological diseases, has announced that the U.S Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation for its gene therapy candidate, AVXS-101, for the treatment of spinal muscular atrophy (SMA).

AVXS-101 is a gene therapy candidate developed for the one-time treatment of SMA type 1 and is the only gene therapy in development for SMA. Following the announcement, the company’s share has increased 13%. The present Breakthrough Therapy Designation is based on preliminary clinical results from the ongoing phase 1 trial of AVXS-101, conducted in collaboration with The Research Institute at Nationwide Children’s Hospital and The Ohio State University. FDA’s Breakthrough Therapy designation is a process designed to accelerate the development and review of drugs that are intended to treat a serious condition.

The trial is designed as an open-label, dose escalation study to determine the safety and efficacy of gene transfer in SMA type 1 patients. The procedure involves intravenous injection of AVXS-101 through a peripheral limb vein and short-term safety will be evaluated over a period of two years. The trial is expected to complete in December 2017.

SMA is a severe neuromuscular disease characterized by the loss of motor neurons leading to progressive muscle weakness and paralysis. It results from a genetic defect in the SMN1 gene which codes for the survival motor neuron (SMN) protein, and affects all muscles in the body. There is no effective treatment for SMA and current drug therapy has been unsuccessful in stabilizing or reversing this disease. The incidence of SMA is approximately one in 10,000 live births. Type 1 SMA, its most severe form, is a lethal genetic disorder characterized by motor neuron loss and muscle deterioration, resulting in mortality or the need for permanent ventilation support before the age of two for greater than 90 percent of patients. SMA Type 1 is one of the leading genetic cause of infant mortality.

Sean P. Nolan, president and CEO of AveXis 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.”

Source: AveXis nabs breakthrough tag for gene therapy, plans pivotal trial start in 2017; Press Release