FDA Approves $3.2 Million Gene Therapy for Duchenne Muscular Dystrophy

USA Today reported that the Food and Drug Administration authorized the initial genetic treatment for Duchenne muscular dystrophy or DMD. This degenerative condition typically renders boys unable to walk around 10 and frequently results in death in their teens or 20s. 

According to the FDA, the biotech company Sarepta Therapeutics medication must demonstrate in a future clinical trial that it enhances physical function and mobility in people with Duchenne muscular dystrophy to remain on the market. According to the accelerated approval procedure, which approves medications for urgently needed illnesses based on evidence that they are likely to have clinical benefits, the therapy was approved. 

It was the first time a therapy of this kind, which delivers a working copy of a gene to replace one that causes disease in a single treatment, had been approved under the accelerated approval framework. The decision was made in response to moving testimonies from families at last month’s FDA advisory committee meeting.  

Progressive muscle weakness brought on by Duchenne muscular dystrophy can cause children to lose their ability to walk by the time they are teenagers, and many don’t survive through their 30s. An estimated one in 3,300 boys is affected. The Elevidys medication, according to Sarepta, will set patients back $3.2 million each. It is intended to be consumed only once. 

Advocates anticipate that, despite its current limitations, this approval will usher in a series of gene therapies that will address the fundamental causes of the condition, which affects between 12,000 and 15,000 children and adults in the USA and about 300,000 people globally. The X chromosome contains the dystrophin gene. Females often have two X chromosomes, allowing the other to make up for a defective X chromosome. Because males cannot adapt due to having just one X chromosome, they are far more prone to developing Duchenne’s. 

According to Brian Abrahams, an analyst with Wall Street company RBC Capital Markets, the age bracket for whom the medicine was approved—children aged 4 and 5—represents around 6% of those with Duchenne muscular dystrophy. He had predicted that Sarepta would charge $2.8 million for each patient, which could generate $2.1 billion in sales from the initial pool of patients eligible for the medication. 

Although outrageous, such a price is comparable to other one-time gene therapies, which have recently cost more than $3 million per patient. But following the $3.5 million hemophilia medicine Hemgenix, Elevidys will overtake it as the costliest medication in the world. 

The FDA’s Centre for Biologics Evaluation and Research director, Dr. Peter Marks, stated in a news release that the approval “addresses an urgent unmet medical need and is an important advancement in the treatment of Duchenne muscular dystrophy, a devastating condition with limited treatment options that causes a progressive deterioration of an individual’s health over time.”

“The FDA remains committed to supporting the development of novel, effective therapies to lessen the effects of disabling illnesses and to enhance treatment outcomes and quality of life for those affected,” the FDA stated. According to the company’s news release, the first outcomes from Sarepta’s confirmatory trial of the medicine are anticipated later this year.