Our response to Sarepta announcement
By now, many of you will already have seen the terrible news, that a young man who was treated with ELEVIDYS, a Duchenne muscular dystrophy (DMD) gene therapy drug owned by Sarepta, has very tragically passed away.
This is awful news and we send our condolences to his family.
ELEVIDYS
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.
What has been announced
Sarepta has said that the young man died from acute liver failure. Acute liver injury is a known possible side effect of ELEVIDYS and other AAV-mediated gene therapies.
Although it is not a new safety signal, acute liver failure (ALF) leading to death represents a severity of acute liver injury not previously reported for ELEVIDYS, which to date has been used to treat more than 800 patients in clinical trials or as a prescribed therapy.
Sarepta said that testing revealed that the young man had a recent cytomegalovirus (CMV) infection which was identified by the treating physician as a possible contributing factor. CMV can infect and damage the liver, a condition known as CMV hepatitis.
ELEVIDYS in the UK
Sarepta will not be applying in the UK to get ELEVIDYS approved. Instead it has licensed the drug outside the US to a European company called Roche, who will be in charge of ELEVIDYS in the UK. Roche will need to apply to the UK’s drug approval body, the Medicines and Healthcare products Regulatory Agency (MHRA).
Roche has previously indicated that it is engaging with the MHRA, and we will contact Roche to find out whether this news affects its plans to seek regulatory approval in the UK.
ELEVIDYS in the US
ELEVIDYS is available as a treatment in the US. The Food and Drug Administration (FDA) in the United States (US) gave conditional approval to ELEVIDYS in June 2023 for the treatment of ambulatory (walking) paediatric patients aged four years and older. It then gave full approval for it to be prescribed to DMD patients who are aged four years and older and non-ambulatory in June 2024.
Understanding gene therapy as a treatment for DMD
If you would like to know more about ELEVIDYS and gene therapy, we held a webinar discuss the results of the EMBARK trial of ELEVIDYS. It was led by Professor Francesco Muntoni, Professor of Paediatric Neurology at Great Ormond Street Hospital. We ran it jointly with the NorthStar Network of DMD specialists, Muscular Dystrophy UK and Action Duchenne.
You can watch it here.
We know this is awful news for our community but we are here for you. If you have any questions about ELEVIDYS or gene therapy for DMD, you can email us at [email protected].