On May 24, 2019, La Jolla, CA-based AveXis, a subsidiary of Novartis, received U.S. Food and Drug Administration (FDA) approval for Gene Thereapy Drug for Spinal Muscular Atrophy.

The medicine, sold by the Swiss drugmaker Novartis, is a gene therapy that treats an inherited condition called spinal muscular atrophy. The treatment targets a defective gene that weakens a child’s muscles so dramatically that they become unable to move, and eventually unable to swallow or breathe. It strikes about 400 babies born in the U.S. each year. The treatment was priced at $2.125 million.