Regenxbio to Receive $80 Million Milestone Payment from Novartis

Regenxbio announced that it will receive a milestone payment of $80 million from Novartis based upon the achievement of $1 billion in cumulative net sales of Zolgensma. Regenxbio expects to recognize this revenue in the third quarter of 2020.

“Zolgensma is a transformative, one-time gene therapy for the treatment of pediatric patients with SMA, a debilitating and potentially deadly disease, and the first approved gene therapy based on Regenxbio’s NAV Technology. We believe that this achievement provides further validation of the broad potential of NAV Technology to improve the lives of patients in need,” Kenneth T. Mills, President and Chief Executive Officer of Regenxbio, said in a company news release. “We are pleased to have contributed to a therapy that has impacted the lives of over 700 patients with SMA and their families around the world.”

Zolgensma uses Regenxbio’s proprietary NAV AAV9 vector and was designed and developed by AveXis, Inc., now known as Novartis Gene Therapies. In May 2019, Zolgensma was granted FDA approval for the treatment of spinal muscular atrophy (SMA) in pediatric patients who are less than 2 years of age, and has since been approved in Europe, Japan and other countries. Regenxbio continues to receive tiered royalties on sales of Zolgensma. Since FDA approval in 2019, Regenxbio has earned more than $140 million in revenues from royalties and commercial milestone payments related to Zolgensma through the third quarter of 2020.