Label for DMD gene therapy Elevidys expanded in the US
The Food and Drug Administration (FDA) in the United States (US) has announced that it has expanded the label for the Duchenne muscular dystrophy (DMD) gene therapy treatment Elevidys.
This follows it granting the treatment, which has been developed by the pharmaceutical company Sarepta, accelerated approval last year.
Expanded label in US
The FDA gave conditional approval to Elevidys in June 2023 for the treatment of ambulatory (walking) paediatric patients aged four years and older. It has now given full approval for it to be prescribed to DMD patients who are aged four years and older and non-ambulatory.
Gene therapy to treat DMD
Elevidys (also known as delandistrogene moxeparvovec, or dela mox, and SRP-9001) is an adeno-associated virus (AAV) vector-based gene therapy product for ambulatory patients with a confirmed mutation in the DMD gene.
It is a one-time treatment designed to treat the underlying cause of DMD.
Availability in UK
The announcement only applies in the US. Britain’s drug approval body, the Medicines and Healthcare products Regulatory Agency (MHRA), will make a decision separately based upon an application by Roche, the company with the licence for Elevidys in the UK.
Information about the approval is available on the FDA’s website.