Despite this promising potential, Elevidys has faced significant challenges. In March 2025, a teenager died from acute liver failure following treatment with Elevidys. A second fatality occurred in June, 2025, involving another non-ambulatory patient who suffered fatal liver complications. Both incidents have raised serious safety concerns, particularly regarding the risk of acute liver injury from the gene therapy.
In response to these events, Sarepta Therapeutics has paused shipments of Elevidys to non-ambulatory patients and halted clinical trials. The company is collaborating with the FDA to implement enhanced safety protocols, including the potential use of immunosuppressants to mitigate immune responses. Additionally, Sarepta has withdrawn its 2025 revenue projections, which had previously claimed over $2 billion in sales, reflecting the uncertainty surrounding Elevidys’ future in the market.
This approach puts already vulnerable children at further risk and prioritizes the rescue and reputation of an experimental pharmaceutical genetically engineered product over safeguarding patient lives.
When a treatment results in acute liver failure and death in multiple children, the only ethical and scientific action is to halt its use entirely until the mechanisms of harm are fully understood; not to layer on more drugs in an attempt to control a known toxicity.
These developments have not only impacted Sarepta’s financial standing, but have also have generated debates within the biotechnology industry and regulatory agencies. The FDA’s decision to grant accelerated approval for Elevidys, despite concerns over its efficacy and safety, has been a point of contention, especially following the Phase 3 trial’s failure to meet their own goals. The recent leadership changes at the FDA have further complicated the regulatory landscape for gene therapies.
As of now, shockingly, Elevidys remains approved for certain patient populations, but its availability and future use are under review. The ongoing investigations and safety assessments will determine whether the therapy can continue to be offered to patients with Duchenne muscular dystrophy.
The tragic saga of Elevidys is an indictment of the reckless rush to embrace genetic engineering as the next frontier of medicine. Far from being the miracle it’s marketed to be, this technology is neither precise nor predictable. It is a high-stakes gamble with human lives.
The deaths of children from liver failure after receiving this so-called therapy should have sent shockwaves through the medical establishment. Instead, we see attempts to cover the damage with more drugs and revised protocols, rather than confronting the root problem: an unproven, unstable technology that is being deployed with a lack of understanding, oversight, or accountability.
Whether it is used in agriculture, vaccines, or medical interventions, genetic engineering is forging ahead without the base knowledge or ethical framework to justify its use, especially in our most vulnerable populations. The lack of any regulation and the blind faith in innovation over safety is not science; it’s exploitation masquerading as progress.