Latest news with #Elevidys
Epoch Times
18 hours ago
- Health
- Epoch Times
Second Patient Dies After Gene Therapy for Duchenne Muscular Dystrophy: Company
A second patient has died after receiving the gene therapy Elevidys, which was approved by the Food and Drug Administration (FDA) over concerns of staffers, according to the company that makes it. The 15-year-old boy who died, and the 16-year-old boy whose death was
Forbes
19 hours ago
- Health
- Forbes
Second Patient Death After Gene Therapy: Implications For The Future
As we continue our series exploring genetic medicine, both in this story and in Destiny's Child No Longer: Rewriting Genetic Fate, gene therapy stands at a crossroads. On June 16, Sarepta Therapeutics announced a second patient death following treatment with its gene therapy, Elevidys, for Duchenne muscular dystrophy. This news has sent shockwaves through the rare disease and biotech communities. It raises questions about the safety of adeno-associated virus-based gene therapies. The latest fatality, a 15-year-old, died from acute liver failure, a recognized but rare complication of adeno-associated virus-based gene transfer. This death follows a similar case reported in March. These recent tragedies have profound implications. Elevidys, Sarepta's gene therapy, was among the first to receive approval for the treatment of Duchenne muscular dystrophy. Yet, the deaths of two patients treated with Elevidys now force us to confront a difficult question. Do the potential benefits of this therapy truly outweigh the risks, particularly for those who can no longer walk and may be at higher risk of complications? Every advance in medicine brings new challenges, and gene therapy is no exception. The deaths associated with Elevidys have raised the regulatory bar for all investigational gene therapies that use adeno-associated virus vectors. This means more stringent safety monitoring, more careful patient selection, and better reporting of outcomes. This heightened scrutiny is not unwelcome, even if these measures may slow the pace of development in the short term. They are essential for ensuring safety and responsibility. Adeno-associated virus-based vectors have become the workhorse of gene therapy due to their efficiency and relatively low risk of complications. The field has long recognized that high-dose adeno-associated virus-based administration can trigger severe, sometimes fatal, immune-mediated toxicities. These primarily affect the liver. In 1999, Jesse Gelsinger died after receiving an adenoviral gene therapy. His tragic death marked a turning point in the field and led to decades of increased scrutiny and regulation. While adeno-associated viruses are generally less immunogenic than adenoviruses, high doses can activate the body's immune system. This leads to inflammation, liver injury, and, in rare cases, organ failure. Recent studies highlight that immune responses can contribute to toxicity, particularly in the liver. However, not all patients are impacted equally. Recent findings underscore that age and disease status can significantly influence a patient's response to adeno-associated virus-based gene therapy. Clinical studies reveal that individual factors, such as underlying infections, pre-existing liver disease, or genetic predispositions, can amplify the risks. For example, in the first reported Elevidys death, a concurrent infection may have played a role in the patient's rapid decline. Research from the University of Bristol indicates that in ocular gene therapy trials, older patients, particularly females, are more likely to experience severe inflammatory responses. However, in the Elevidys cases, both fatalities involved males who had lost the ability to walk due to advanced disease progression. This is a critical factor linked to heightened immune vulnerability. This underscores that risks are context-specific, depending on therapy type, delivery site, and patient health status. To address the pressing safety concerns, Sarepta is investigating several strategies. One approach under consideration is the addition of sirolimus, an immunosuppressant widely used in organ transplantation. While this aims to reduce immune-mediated liver damage, experience from liver transplant patients warns that sirolimus can nearly triple infection risk, even at low doses. Its use in gene therapy remains experimental and demands careful evaluation before it can be widely adopted. At the same time, non-viral delivery methods are emerging as a promising alternative. Lipid nanoparticles offer a way to bypass many of the immune challenges posed by viral vectors. Their ability to be tailored for precise tissue targeting and the absence of pre-existing immunity in patients make them particularly attractive for conditions like Duchenne muscular dystrophy, where repeated dosing may be necessary. Though still less efficient than viral vectors in some applications, the technology is advancing quickly. The second death following Elevidys for Duchenne muscular dystrophy is a sobering reminder of the risks inherent in adeno-associated virus-based gene transfer, particularly for older or more severely affected patients. Still, these tragedies are not new to the field. We have seen similar inflection points before. The history of gene therapy is marked by moments of triumph and tragedy. Each shapes the path forward. As we reflect on these recent events, it is clear that the field stands at another such crossroads—one that will require careful balancing of hope, caution, and scientific rigor to chart the best course for patients and the future of medicine. For a deeper exploration of these issues, including real-world patient stories and the latest scientific advances, see Destiny's Child No Longer: Rewriting Genetic Fate, which provides a detailed examination of this therapy and other gene therapies.
Business Wire
19 hours ago
- Business
- Business Wire
SRPT BREAKING NEWS: Sarepta Therapeutics, Inc. is being Investigated for Securities Fraud after Second Elevidys Death; Investors with Losses are Notified to Contact BFA Law
NEW YORK--(BUSINESS WIRE)--Leading securities law firm Bleichmar Fonti & Auld LLP announces an investigation into Sarepta Therapeutics, Inc. (NASDAQ: SRPT) for potential violations of the federal securities laws. Leading securities law firm Bleichmar Fonti & Auld LLP announces an investigation into Sarepta Therapeutics, Inc. (NASDAQ: SRPT) for potential violations of the federal securities laws. Share If you invested in Sarepta, you are encouraged to obtain additional information by visiting: Why is Sarepta being Investigated? Sarepta is a biopharmaceutical company focused on developing treatments for rare diseases. Sarepta's most important product is Elevidys, a therapy for the treatment of Duchenne muscular dystrophy. During the relevant period, Sarepta repeatedly touted the safety profile of Elevidys and told investors that the benefits of the treatment outweighed its risks. In truth, Elevidys causes fatal acute liver failure in some patients. The Stock Declines as the Truth is Revealed On March 18, 2025, Sarepta announced that a patient that had been treated with Elevidys died after suffering acute liver failure. On this news, the price of Sarepta stock fell $27.81 per share, or over 27%, from $101.35 per share on March 17, 2025, to $73.54 per share on March 18, 2025. Nevertheless, on the same day, Sarepta assured investors that 'the benefit-risk of ELEVIDYS remains positive.' Next, on June 15, 2025, Sarepta announced that a second patient treated with Elevidys had died from acute liver failure. The company further announced that because of the second death, it was suspending certain shipments of Elevidys and paused dosing in an ongoing clinical trial of the treatment. On this news, the price of Sarepta stock fell $15.24 per share, or more than 42%, from $36.18 per share on June 13, 2025, to $20.94 per share on June 16, 2025. Click here for more information: . What Can You Do? If you invested in Sarepta you may have legal options and are encouraged to submit your information to the firm. All representation is on a contingency fee basis, there is no cost to you. Shareholders are not responsible for any court costs or expenses of litigation. The firm will seek court approval for any potential fees and expenses. Why Bleichmar Fonti & Auld LLP? Bleichmar Fonti & Auld LLP is a leading international law firm representing plaintiffs in securities class actions and shareholder litigation. It was named among the Top 5 plaintiff law firms by ISS SCAS in 2023 and its attorneys have been named Titans of the Plaintiffs' Bar by Law360 and SuperLawyers by Thompson Reuters. Among its recent notable successes, BFA recovered over $900 million in value from Tesla, Inc.'s Board of Directors, as well as $420 million from Teva Pharmaceutical Ind. Ltd. For more information about BFA and its attorneys, please visit Attorney advertising. Past results do not guarantee future outcomes.
Forbes
20 hours ago
- Business
- Forbes
SRPT Stock: What's Happening With Sarepta Therapeutics?
CANADA - 2025/06/08: In this photo illustration, the Sarepta Therapeutics logo is seen displayed on ... More a smartphone screen. (Photo Illustration by Thomas Fuller/SOPA Images/LightRocket via Getty Images) Sarepta Therapeutics (NASDAQ: SRPT) suffered a devastating blow on Monday, June 16 when its stock plummeted 42% following news of a second patient death linked to its Duchenne muscular dystrophy gene therapy, Elevidys. The biotechnology company and its global partner Roche immediately suspended commercial and clinical use of the treatment in non-ambulatory patients pending a comprehensive safety review. The decision came after Roche conducted a safety profile reassessment that revealed concerning patterns following the two fatalities. Commercial distribution has ceased effective immediately, while enrollment in ongoing clinical trials has been paused until additional risk mitigation measures can be implemented in study protocols. Now, if you are looking for an upside with a smoother ride than an individual stock, consider the High-Quality portfolio, which has outperformed the S&P, and clocked >91% returns since inception. On a separate note, see – Should You Buy CRWV Stock After A Whopping 4x Rise? Elevidys represents a significant advancement in gene therapy for Duchenne Muscular Dystrophy, a devastating genetic disorder that progressively weakens muscles. The FDA granted accelerated approval in June 2023 for ambulatory patients aged 4-5, later expanding traditional approval to all ambulatory patients aged 4 and older. However, continued approval for non-ambulatory patients remained contingent on confirmatory clinical trial results. The financial implications for Sarepta are profound. Elevidys generated 43% of the company's total revenues last year, making it the cornerstone of Sarepta's commercial portfolio. Industry analysts had projected peak sales of over $3 billion for the therapy, expectations that now face substantial revision given the restricted patient population. Sarepta's financial profile already presented significant challenges before this setback. Despite achieving average revenue growth exceeding 40% over the past three years, the company remains unprofitable with an operating cash flow margin of negative 25% and a net income margin of negative 11%. The company carries $1.3 billion in debt against a current market capitalization of $2.06 billion, resulting in a concerning debt-to-equity ratio of 63%. The stock's trajectory illustrates the extreme volatility inherent in pharmaceutical investments. Shares have collapsed from their 52-week high of approximately $172 to current levels around $20, representing the kind of dramatic price swings that characterize companies heavily dependent on revenues from only a few drugs. While Sarepta's multi-year low valuations might appear attractive to contrarian investors, the investment proposition carries substantial risks. The combination of recent safety concerns with its flagship product, overwhelming dependence on a single revenue source, significant cash burn, and extreme stock volatility creates a highly speculative investment scenario suitable only for investors with high risk tolerance. Investors seeking exposure to innovative healthcare developments might consider diversified approaches, such as the Trefis High Quality (HQ) Portfolio, that spread risk across multiple companies and sectors, rather than concentrating capital in individual speculative positions. With a collection of 30 stocks, this portfolio has a track record of comfortably outperforming the S&P 500 over the last 4-year period. Why is that? As a group, HQ Portfolio stocks provided better returns with less risk versus the benchmark index; less of a roller-coaster ride, as evident in HQ Portfolio performance metrics.
Time of India
21 hours ago
- Health
- Time of India
Second patient death reported with gene therapy for muscular dystrophy
Washington: Shares of Sarepta Therapeutics plunged Monday after the biotech drugmaker reported a second death in connection with its gene therapy for muscular dystrophy . Sarepta reported the death over the weekend and provided additional details about its response, which includes pausing shipments of the therapy, Elevidys, for older patients who are no longer able to walk. The one-time treatment is approved for children with a genetic variant of Duchenne's muscular dystrophy, which causes weakness, loss of mobility and early death in males. Elevidys is the first gene therapy approved in the U.S. for the rare muscle-wasting condition, but it has faced scrutiny since its accelerated approval in 2023. The second death, like an earlier one reported in March, occurred in a teenage boy who suffered a fatal case of acute liver injury, a known side effect of the therapy. Older patients receive a larger dose of the therapy. Sarepta said it would pause a study in those patients and assemble an expert panel to recommend new safety protocols for taking the drug. Those changes are expected to include increased use of immune-system suppressing drugs, company executives said Monday. The liver injury associated with the therapy is thought to be connected to the immune system's response. Sarepta said it was cooperating with the Food and Drug Administration, which would have to sign off on any changes to the product's use. Elevidys received expedited approval despite concerns from some FDA scientists about its effectiveness in treating Duchenne's. The FDA granted full approval last year and expanded the therapy's use to patients 4 years and older, regardless of whether they are still able to walk. Previously it was only available for younger patients who were still walking. Shares of the Cambridge, Massachusetts-based company fell more than 42% to close at about $21 in trading. Wall Street analysts speculated that FDA officials, including new vaccine chief Dr. Vinay Prasad, might impose more restrictions on the drug or even block its use. Prasad has been highly critical of the therapy since its approval under the agency's previous leadership. "Now with two deaths reported in this segment of the market, it seems incrementally more possible that the FDA could step in and remove the therapy from the market in non-ambulatory patients," said Leerink Partners analyst Joseph Schwartz, in a Sunday research note. Elevidys uses a disabled virus to insert a replacement gene for producing dystrophin into patient cells. It costs $3.2 million for a one-time infusion.
