AveXis Receives Orphan Drug Status from the US Food and Drug Administration (FDA) for chariSMA for the Treatment of Spinal Muscular Atrophy
Summary
These unfortunate infants are born with a mutated or malfunctioning gene on the 5th chromosome, reducing production of a key protein called SMN which stands for survival motor neuron.SMN is required in motor neurons that control muscles that are involved in breathing, sitting up and swallowing, without it these children pass away, it is my hope that the incredible pre-clinical work pioneered by Dr. Brian Kaspar and others will translate into humans,” said John A. Carbona, Chief Executive Officer of AveXis.Dallas, TX, October 04, 2014 --( PR.com )-- AveXis Inc., a synthetic biology platform company primarily focused on developing treatments for Spinal Muscular Atrophy (SMA) today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to its product scAAV9, called chariSMA to treat Spinal Muscular Atrophy patients.SMA is an autosomal recessive birth defect, and the largest genetic killer of infants.These unfortunate infants are born with a mutated or malfunctioning gene on the 5th chromosome, reducing production of a key protein called SMN which stands for survival motor neuron.SMN is required in motor neurons that control muscles that are involved in breathing, sitting up and swallowing, without it these children pass away, it is my hope that the incredible pre-clinical work pioneered by Dr. Brian Kaspar and others will translate into humans,” said John A. Carbona, Chief Executive Officer of AveXis.AveXis is also working with leading researches in European centers to understand the financial and regulatory pathways forward to initiate a mirror image trial in Europe in 2015.The Orphan Drug designation is granted to development-stage novel therapeutics that offer potential value in the treatment of rare diseases and medical conditions that affect fewer than 200,000 patients in the US.