FDA expands approval of first gene therapy for rare form of muscular dystrophy
How much does one dose of Elevidys cost?
One dose of Elevidys costs approximately $3.2 million per patient. This price tag makes it the second most expensive drug in the world, after the $3.5 million hemophilia treatment Hemgenix.
What is Elevidys used to treat?
Elevidys is a gene therapy used to treat Duchenne muscular dystrophy (DMD) in ambulatory and non-ambulatory individuals 4 years of age and older with a confirmed mutation in the DMD gene1. It is designed to deliver a gene that leads to the production of micro-dystrophin, a shortened protein that contains selected domains of the dystrophin protein present in normal muscle cells6.
What is the primary genetic mutation targeted by Elevidys?
Elevidys targets the genetic root cause of Duchenne muscular dystrophy (DMD) by introducing an engineered gene that codes for a shortened version of dystrophin, referred to as ELEVIDYS micro-dystrophin, into muscle cells6. The primary genetic mutation targeted is in the DMD gene, which is associated with muscle strength.