Gene Therapy for Spinal Muscular Atrophy approved by FDA

A new treatment for a rare childhood disorder which cost $2.125 million for a single dose, one of the most expensive medicine available is approved by The US Food and Drug Administration.

Spinal Muscular Atrophy, a condition driven by defects in the SMN1 gene causing burdened babies lose muscle control, the medicine is designed to treat the disease. In the US each year about 400 babies is being affected by the illness and kills those with the most common type of the disease in a couple of years. The gene therapy treatment uses genetically modified viruses to deliver health copies of the SMN1 gene to patients’ cells so they can produce a protein that enables the children to grow normally.

In trial of the treatment, babies who got it by 6 months of age didn’t have as serious muscle issues as those who didn’t get the medication. New-borns getting the drug after 6 months, didn’t lose muscle control, however they endured irreversible damage harm. According to Associated Press, those babies were the healthiest, who got the treatment early.

David Lennon, President of AveXis, the company, owned by Novartis that developed the drug, called Zolgensma, said “We saw just remarkable results for these kids and it is only the second FDA-approved gene therapy designed to treat genetic disorder.