There is currently no cure for Duchenne muscular dystrophy (DMD). Treatments, such as physical therapy and the use of oral corticosteroids, are designed to help manage symptoms and improve a patient’s quality of life.
Elevidys is a new gene therapy designed to address the root cause of DMD by delivering treatment to muscle cells to improve muscle function. Although many are excited about the hope Elevidys offers, others are concerned that the drug is not worth the risks that come with its use. To date, two patients who received Elevidys therapy have died from acute liver failure.
If the use of Elevidys has harmed you or your loved one, you may be entitled to recover compensation. Dangerous drug claims are complex. You need the help of a Chicago wrongful death attorney with a proven record of success handling drug injury claims.
At Meyers & Flowers Trial Attorneys, we have that proven record. We have helped recover billions of dollars in compensation for those injured by dangerous drugs, and we want to help you recover maximum compensation, too.
What Is Duchenne Muscular Dystrophy (DMD)?
According to the Muscular Dystrophy Association (MDA), Duchenne muscular dystrophy (DMD) is a progressive, genetic neuromuscular disorder caused by a mutation in the DMD gene, which results in the dysfunction or absence of the dystrophin protein. The dystrophin protein helps maintain the health of muscle cells. Without that protein, muscles become weak and vulnerable to injury.
DMD occurs in approximately one out of every 3,500 male births, and its symptoms appear in early childhood. Although DMD primarily affects males, females may be symptomatic carriers, and in some cases, females are severely affected.
This condition causes skeletal and cardiac muscle weakness that worsens rapidly over time, according to the Cleveland Clinic. DMD symptoms can severely affect a person’s quality of life and shorten life expectancy. Available treatments focus on slowing its progression and managing symptoms.
What Kind of Drug Is Elevidys, And Who Manufactures It?
Elevidys is a new one-time gene therapy used to help maintain muscle strength in patients with DMD. It is manufactured by the pharmaceutical company Sarapta Therapeutics. In 2023, the Food and Drug Administration (FDA) granted accelerated approval based on clinical trials that suggested Elevidys increases muscle strength and mobility in patients.
Critics argue that the FDA approval is suspect because two clinical trials found that the drug was not significantly better than a placebo.
In a June 22, 2023, news release, the FDA’s director of the Center for Biologics Evaluation and Research (CBER), Peter Marks, stated:
“Today’s 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 leads to a progressive deterioration of an individual’s health over time. 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.”
Interestingly, as the CBER director, Peter Marks overruled the agency’s internal review team on two occasions for Elevidys to receive accelerated approval. Peter Marks no longer serves as the CBER director. The new director of the CBER is Vinay Prasad, who criticized the approval of Elevidys when Peter Marks was the director.
How Does Elevidys Work?
Elevidys utilizes a tool known as a vector to deliver a shortened version of the dystrophin gene, referred to as micro-dystrophin, to the muscles. The micro-dystrophin signals the body to produce more dystrophin. When the body produces more dystrophin, muscles can stay stronger for longer.
Does Elevidys Cause Liver Failure?
As of this writing, two patients who received Elevidys injections have recently died from liver failure. Liver damage is a known risk factor associated with gene therapies.
The Elevidys label currently lists acute liver injury in its Warnings and Precautions section. Sarepta may be updating its label in light of the two deaths due to acute liver failure.
Both patients who died were non-ambulatory and no longer able to walk without assistance due to the progression of DMD. Since the second death, Sarapta Therapeutics has suspended Elevidys treatment to non-ambulatory patients in the United States.
It is now seeking FDA approval to use a new drug in conjunction with Elevidys to help manage liver toxicity. Sarapta wants to amend the trial protocol of Elevidys to add the drug sirolimus to the therapy. Sirolimus has been previously used in other gene therapies to help control increases in liver enzymes.
While Sarapta’s suspension of treatment and proposed use of sirolimus only apply to non-ambulatory patients, some analysts question whether these conditions should also apply to ambulatory patients.
Patients have been delaying treatment since learning of the deaths of two Elevidys recipients, and Sarepta’s stock price has plummeted due to the lower sales outlook for the drug.
A class action lawsuit has now been filed against Sarepta Therapeutics, Inc. for securities purchased between June 22, 2023, and June 24, 2025. The lawsuit complaint alleges that Sarepta:
- Misrepresented the safety of Elevidys, despite serious risks;
- Failed to detect or disclose severe adverse events during clinical trials;
- Withheld material information that eventually led to halted dosing, increased regulatory scrutiny, and suspension of shipments; and
- Lacked a reasonable basis for positive statements about Elevidys’s safety and potential for expanded use.
What To Do If You Have Been Injured By Elevidys Or Other Dangerous Drugs
When therapies like Elevidys harm the patients they were supposed to help, victims may have legal options for compensation. Dangerous drug suits may be filed against the drug’s manufacturer, distributor, or seller. Such legal claims are typically based on product liability laws that hold companies accountable for selling unsafe or defective products.
Class action lawsuits are often used to sue pharmaceutical companies for damages resulting from a defective drug. However, not every victim experiences the same harm, and class actions are not always the best choice.
Patients and families who have suffered harm caused by Elevidys or other drugs need the help of an experienced dangerous drug attorney to help them understand their options and protect their rights.
The Chicago dangerous drug lawyers at Meyers & Flowers have intimate knowledge of pharmaceutical company practices and the FDA approval process. We know how to investigate dangerous drug claims to help identify and gather essential evidence and build your strongest case.
Contact an Experienced Dangerous Drug Lawyer Today
The Chicago medical malpractice lawyers at Meyers & Flowers have decades of experience protecting the rights of injured patients and their families and have recovered billions of dollars in client compensation. We have the knowledge, skills, and resources to help you recover maximum compensation for your damages.
Call (630) 895-9554 or complete the short form on our contact page to schedule a free consultation to discuss your dangerous drug claim.