AveXis Gains Priority Review for Rare Pediatric Disorder Drug
The FDA granted AveXis a priority review voucher for its gene therapy candidate Zolgensma (onasemnogene abeparvovec-xioi), for treatment of spinal muscular atrophy in newborns.
Zolgensma is approved by the FDA to treat pediatric patients less than two years of age who have spinal muscular atrophy with biallelic mutations in their survival motor neuron 1 gene.
Spinal muscular atrophy results in the deterioration of motor neuron cells that directly and indirectly control organs. The disease saps the physical strength of patients until they are eventually unable to walk, eat or breathe.