​FDA approves REGENXBIO gene therapy for pediatric spinal muscular atrophy


The FDA approved REGENXBIO’s (NASDAQ:RGNX) ZOLGENSMA for the treatment of spinal muscular atrophy (SMA) in pediatric patients who are less than two years of age.

Caused by a genetic defect in the SMN1 gene, SMA is characterized by the loss of motor neurons, leading to progressive muscle weakness and paralysis.

ZOLGENSMA uses REGENXBIO’s proprietary NAV gene delivery platform to transfer a functional SMN1 gene to the patient via a one-time infusion.

“ZOLGENSMA’s approval demonstrates the transformational impact that NAV technology can have on the treatment of genetic diseases with significant unmet needs,” Kenneth Mills, REGENXBIO’s president and CEO, said in a statement.

“ZOLGENSMA is the first of many NAV technology gene therapy product candidates in our research and clinical development pipeline with the potential to treat a large range of genetic and chronic diseases,” he added.

The approval triggers a $3.5-million milestone payment from development partner, AveXis, a Novartis company. ZOLGENSMA is under regulatory review in Europe and Japan, and REGENXBIO anticipates approval in these jurisdictions in 2019.

Jennifer Poland