The FDA has granted a breakthrough therapy designation to AVXS-101 (AveXis, Inc.) for the treatment of spinal muscular atrophy (SMA) type 1 in pediatric patients. AveXis is a clinical-stage gene therapy company developing treatments for patients suffering from rare and life-threatening neurological genetic diseases.
The breakthrough therapy designation is based on preliminary clinical results from the ongoing trial of AVXS-101, conducted in collaboration with The Research Institute at Nationwide Children’s Hospital and The Ohio State University.
“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,” said Sean P. Nolan, President and Chief Executive Officer of AveXis. “By this action, the FDA recognizes the high unmet need for effective treatment options for patients suffering from SMA.”
The FDA and the company are expected to schedule a multidisciplinary, comprehensive discussion of the development program for AVXS-101.
SMA is a severe neuromuscular disease characterized by the loss of motor neurons leading to progressive muscle weakness and paralysis. SMA is caused by a genetic defect in the SMN1 gene that codes SMN, a protein necessary for survival of motor neurons. The incidence of SMA is approximately one in 10,000 live births.
The most severe form of SMA is type 1, a lethal genetic disorder characterized by motor neuron loss and associated muscle deterioration, which results in mortality or the need for permanent ventilation support before the age of 2 years for greater than 90% of patients. SMA type 1 is the leading genetic cause of infant mortality.
AVXS-101 is a proprietary gene therapy candidate for a one-time treatment for SMA type 1 and is the only clinical-stage gene therapy in development for SMA. AVXS-101 is designed to address the monogenetic root cause of SMA and prevent further muscle degeneration by addressing the defect in and/or loss of the primary SMN gene. AVXS-101 also targets motor neurons providing rapid onset of effect and crosses the blood–brain barrier, allowing an intravenous dosing route and effective targeting of both central and systemic features.
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