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FDA Approves First Gene Therapy for Treatment of Certain Patients with Duchenne Muscular Dystrophy

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The FDA has officially granted approval on June 22nd 2023 to Elevidys, marking it as the first gene therapy for addressing Duchenne muscular dystrophy (DMD) in paediatric patients aged 4 to 5. This approval applies specifically to patients with confirmed DMD gene mutations and no medical conditions that would hinder treatment with this therapy.

Sarepta Therapeutics’ gene therapy received authorization under accelerated approval from to treat Duchenne Muscular Dystrophy, this pathway helps the development of drugs for serious or life-threatening diseases when there is an unmet medical need and potential.

“The FDA remains committed to facilitating the development of innovative new therapies to reduce the impact of debilitating diseases and to improve outcomes and quality of life for those affected.”

What is Duchenne muscular dystrophy?

Duchenne muscular dystrophy (DMD) is a genetic disorder characterized by progressive muscle weakness and degeneration, it is caused by mutations in the gene that encodes the protein dystrophin, which is necessary for muscle stability and function as the muscle fibers become weak, damaged and eventually replaced by fat and connective tissue.

As a result of this genetic defect, individuals with DMD may have symptoms such as trouble walking and running, falling frequently, fatigue, learning disabilities/difficulties, heart issues and breathing problems due to weakening of respiratory muscles involved in lung function.  These symptoms associated with Duchenne muscular dystrophy typically begin in childhood, often between 3 to 6 years of age.



Elevidys is a recombinant gene therapy aimed at introducing a specific gene into the body. This gene prompts the production of a modified form of dystrophin called Elevidys micro-dystrophin.

Unlike the full-length dystrophin found in healthy muscle cells (which weighs 427 kDa), the Elevidys micro-dystrophin is a shorter protein, weighing 138 kDa. It includes specific segments of the normal dystrophin protein, and its administration involves a single intravenous dose.


Treatment for Duchenne muscular dystrophy

The FDA approved Elevidys after reviewing the data provided by the sponsor. The evaluation included a study consisting of two parts.

  • In part 1—which was randomized, double-blind, and placebo-controlled— the individuals were treated with either Elevidys or placebo and followed for 48 weeks.
  • In part 2 of the study, individuals who received placebo during part 1 were treated with Elevidys and vice versa for the individuals that were treated with Elevidys. All individuals were followed for an additional 48 weeks.

Available treatments for Duchenne muscular dystrophy (DMD) encompasses the use of corticosteroid medications, exercise regimens and assistive devices like bracers or wheelchairs becomes challenging. Nevertheless, most existing treatment strategies primarily focus on managing the symptoms of the disease rather than directly targeting its root genetic cause.


Side effects of Elevidys

Commonly reported side effects are vomiting, nausea, acute liver injury, pyrexia (fever), thrombocytopenia (abnormally low platelet count in the blood), severe immune-mediated myositis (inflammation of muscle) in patients receiving Elevidys. Clinical trials have also shown instances of myocarditis (inflammation of heart muscle) and elevated levels of troponin-I (a protein released into the bloodstream after heart muscle injury) following the use of Elevidys hence, to mitigate these risks, it is recommended to monitor troponin-I levels prior to administering Elevidys and weekly for the initial month after treatment.


The official FDA press release contains more information if you want to know more or to consult the complete updated information related to the Approval of the First Gene Therapy, you can check it here!

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