The U.S. Food and Drug Administration recently granted ChariSMA, an experimental gene therapy for the treatment of Spinal Muscular Atrophy, orphan drug status, which is given to therapeutic products aimed at treating rare diseases or conditions that affect less than 200,000 people in the United States. ChariSMA is being developed by Dallas-based AveXis, and is currently in phase 1 clinical trials at Nationwide Children’s Hospital in Columbus, Ohio.
The orphan drug designation granted to ChariSMA by the FDA Office of Orphan Drug Products enables novel treatments in early stages of development with potential value in treating rare diseases to be eligible for several incentives outlined in the Orphan Drug Act, including tax credits for qualified clinical testing and market exclusivity.
“SMA is a devastating disease for everyone it touches. Our technology is very simple to describe,” explained the Chief Executive Officer of AveXis, John A. Carbona. “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.”
ChariSMA is an intravenous therapy that delivers a genetically-modified virus, which is comprised of a replacement gene called Survival Motor Neuron gene (SMN), through AveXis’s technology ReGenX Biosciences. AveXis recently closed a financing round, from PBM Capital Group, a private investment firm focused on healthcare and life science investments, which allowed them to fund the beginning of the clinical studies for ChariSMA.