What is Elevidys prescribed for?
To treat Duchenne muscular dystrophy (DMD) in patients who are 4 years of age and older and have a confirmed mutation in the DMD gene.
What is the name of the drug and what does it do?
Elevidys (generic name: delandistrogene moxeparvovec-rokl), pronounced “el-eh-vid-iss,” is a special treatment approved by the U.S. Food and Drug Administration to help with Duchenne muscular dystrophy (DMD).
It works by fixing the genetic problem that leads to DMD. This treatment delivers a gene that makes a smaller protein called micro-dystrophin. This protein is important for keeping muscle cells strong and healthy.
When someone has DMD, their body doesn’t make enough of the dystrophin protein, which is needed for muscles to work properly.
By giving the body the gene to make micro-dystrophin, Elevidys aims to help improve muscle function in people with DMD. It’s like giving the body a tool it needs to strengthen the muscles affected by this condition.
How does it work?
Duchenne muscular dystrophy is a rare genetic condition that leads to muscle weakness and wasting due to a defective gene affecting the production of dystrophin, a protein crucial for muscle cell health.
Elevidys works by introducing a gene that produces micro-dystrophin, a smaller version of the dystrophin protein, into the body.
This therapy aims to improve muscle function in individuals with DMD.
What did the research discover?
The FDA approved Elevidys for Duchenne muscular dystrophy (DMD) after studying 218 male patients with confirmed DMD gene issues.
They looked at how well Elevidys worked in these patients through different types of studies.
Even though one big study didn’t show a clear improvement in a specific test they did, some other results were promising. They found that Elevidys helped with muscle function, strength, and certain blood markers.
Elevidys got the green light for ambulatory DMD patients aged 4 and up, except for those with specific gene deletions. It also got a quicker approval for non-ambulatory patients aged 4 and up.
This was based on data showing that higher levels of a specific protein linked to better outcomes.
Safety data from 156 patients showed common side effects like vomiting and liver issues. Doctors need to watch patients’ liver and heart health closely during treatment.
What are some of the side effects?
The side effects listed are from the product manufacturer. As health scientists, we share this information to keep you informed without causing undue worry. Many side effects diminish over time, and not all patients experience them. If prescribed medication, take it as directed and consult your healthcare professional if you have any concerning side effects.
- Vomiting
- Nausea
- Acute Liver Injury
- Fever
- Thrombocytopenia: This condition involves a decrease in the number of platelets in the blood, which can lead to issues with blood clotting and bleeding.
- Immune-Mediated Myositis: Elevidys may trigger severe muscle inflammation known as myositis, due to the immune system trying to fight off the drug.
- Myocarditis: Inflammation of the heart muscle, known as myocarditis
- Elevations of Troponin-I: Troponin-I is a protein found in the blood that signals heart muscle injury.
What are the dosage recommendations and how is it prescribed?
Elevidys is a single-dose treatment given as an intravenous infusion, with the dose based on body weight at 10 mL per kilogram.
Source:
FDA Expands Approval of Gene Therapy for Patients with Duchenne Muscular Dystrophy [Internet]. U.S. Food and Drug Administration. 2024 [cited 2023 Jun 28]. Available from: https://www.fda.gov/news-events/press-announcements/fda-expands-approval-gene-therapy-patients-duchenne-muscular-dystrophy
