FDA urges Elevidys to pause after third death; Sarepta refuses
Despite a request from U.S. regulators to halt shipments, the company is refusing to pause deliveries for ambulatory patients.
On July 18, the Food and Drug Administration confirmed that it had asked Sarepta to voluntarily stop distributing Elevidys, a gene therapy for Duchenne muscular dystrophy. The request came during a meeting between the agency and the company.
Sarepta declined the FDA's request, stating it would continue shipping Elevidys to patients who are able to walk while maintaining an existing pause for non-ambulatory patients — a halt implemented by the company on June 15 after reporting a case of acute liver failure.
Sarepta said its decision to continue shipping to ambulatory patients was "based on our comprehensive scientific interpretation of the data, which shows no new or changed safety signals in the ambulant patient population."
The most recent patient death — a 51-year-old man with limb girdle muscular dystrophy — did not involve Elevidys but another experimental therapy using similar gene technology. The FDA said clinical trials involving limb girdle muscular dystrophy have now been placed on hold due to safety concerns.
Earlier this year, two teenage boys receiving Elevidys died of acute liver failure. All three deaths occurred in non-ambulatory patients. These events have raised alarm among regulators, investors, and patient advocacy groups.
Elevidys received traditional FDA approval in 2024 for ambulatory patients aged four and older with Duchenne muscular dystrophy. It was also granted accelerated approval for non-ambulatory patients, despite the therapy failing to meet its primary goal in a late-stage trial.
In a further regulatory setback, the FDA also revoked Sarepta's platform technology designation for its AAVrh74 gene therapy — a status that would have streamlined approval for similar treatments.
Following the news of the third death and the regulatory developments, Sarepta shares plunged 36 percent on July 18 to US$14.08, briefly hitting a more than nine-year low.
The FDA is continuing to investigate the risk of acute liver failure linked to Sarepta's platform technology. Some analysts have said this could make patients and families more reluctant to consider the therapy.
"Families with Duchenne muscular dystrophy are grappling with a mix of disappointment, concern... and uncertainty about choices they are making for their children or themselves," said Debra Miller, founder of the non-profit CureDuchenne.
Sarepta said earlier this week that it is working with the FDA to update Elevidys' packaging to include a warning about liver toxicity risks.
However, the company is also facing criticism for its communication handling. During an investor call, analysts questioned why Sarepta had not disclosed the latest patient death earlier in the week when it announced 500 layoffs and financial restructuring. CEO Doug Ingram responded that the death was "neither material nor central" to that announcement.
Some analysts, including those at BMO Capital Markets, have warned that how the disclosure was managed could damage Sarepta's credibility with investors.
Hashtags
Try Our AI Features
Explore what Daily8 AI can do for you:
Comments
No comments yet...
Related Articles
Vancouver Sun
2 hours ago
- Vancouver Sun
Why biohacker Bryan Johnson wants to sell his anti-aging business to build a new religion
Tech entrepreneur and longevity crusader is thinking of ditching his 'pain-in-the-ass' anti-aging nutrition and supplements company to focus on his 'Don't Die' movement, a community of likeminded biohackers 'united in defeating all causes of human and planetary death.' In a lengthy interview with Wired, the 47-year-old California multimillionaire said he is 'so close' to shutting down or selling Blueprint, a wellness company devoted to 'maximally slowing' aging and reversing aging that's already occurred. 'I've been talking to people about this. I don't need the money, and it's a pain-in-the-ass company,' Johnson told Wired's Katie Drummond. Start your day with a roundup of B.C.-focused news and opinion. By signing up you consent to receive the above newsletter from Postmedia Network Inc. A welcome email is on its way. If you don't see it, please check your junk folder. The next issue of Sunrise will soon be in your inbox. Please try again Interested in more newsletters? Browse here. Johnson said Blueprint evolved from his own personal search for a clean, low-in-heavy-metals protein powder into a business venture that was just 'trying to do people a solid. The problem is now people see the business and give me less credibility on the philosophy side. I will not make that trade off … So yeah, I don't want it.' Here's what to know about Johnson, Blueprint, his new religion and why he believes a crude AI copy of Bryan already exists. Johnson, who told Wired he grew up poor ('My mom made my clothes') and remained so until he was 34, made a vast sum after selling his mobile payment processing platform to PayPal for a reported US$800 million, according to Fortune. The self-described most measured human on the planet, Johnson takes 40 odd vitamins and supplements daily as well as hundreds of daily measurements of his heart, liver, lungs, kidneys and other body organs so that they may 'speak for themselves what they need to be in their ideal state.' In 2023, Johnson, his then 17-year-old son and Johnson's father participated in a multi-generational plasma exchange. Johnson received plasma from a litre of blood siphoned from his son at a Texas spa in the hope his son's blood would make him younger. The plasma swap apparently had a null effect: In January, Johnson posted on X that he was no longer injecting his son's blood and had 'upgraded' to another controversial plasma protocol. I am no longer injecting my son's blood. I've upgraded to something else: total plasma exchange. Steps: 1. Take out all blood from body 2. Separate plasma from blood 3. Replace plasma with 5% albumin & IVIG Here's my bag of plasma. Who wants it? 🧵 Johnson eats all the day's food before noon and sticks to a strict high-fibre, 'veggies and legumes,' no alcohol, no sugar diet that makes him feel sharper while avoiding 'post meal dead zones,' he's shared on X. Last year, in a therapy dubbed 'Project Baby Face,' Johnson attempted to restore volume he's lost on his face from a calorie-reduced diet with fat injections in his temple, cheeks and chin. He didn't have enough of is own body fat so he used donor fat. It didn't go well: 'Immediately following the injections, my face began to blow up,' Johnson posted on Instagram . 'And then it got worse, and worse, and worse until I couldn't even see,' a severe allergic reaction. Johnson has explained how, In 2021, 'I endeavoured to figure out proximity to longevity escape velocity. How far away are we from one year of chronological time passing and one staying the same age biologically? I called this Project Blueprint.' He set out with his team to build an algorithm that would help him not die. Blueprint sells 'longevity' supplements, mixes, capsules, powders, snacks, travel packs and biomarker testing kits. In response to a New York Times story on Blueprint published earlier this year, Johnson told Wired the story 'painted it like we are in some kind of emergency financial situation. That is not the case. We are break-even, and I've said that publicly many times. We've had profitable months; we've had loss months. I've been very clear: We priced or products at the exact level to basically be break-even. Additional margin is just not worth it to me.' Does Bryan Johnson think he's going to die? When asked by Wired to respond true or false to the statement that at some point in the future he will no longer be alive, Johnson, who sports a 'broomstick-straight posture and pallid complexion,' as per Men's Health's Jason Rogers, responded in the negative. Humans 'have this real possibility of extending our lifespans to some unknown horizon. So that's extension,' Johnson said. 'But we also have the ability to begin moving ourselves to computational systems. So currently, in a very crude form, I have a Bryan A1 that has digested everything I've ever said.' 'You do currently have this,' Wired asked. 'I do,' Johnson responded. 'And that Bryan A1 is pretty good,' he said. 'When you give birth to superintelligence, you can start extending lifespans to some unknown horizon: 200 years, 1,000 years, 10,000 years. Millions of years. We don't know,' Johnson told Wired. 'When that happens, the entire game of humanity shifts from that singular focus on wealth accumulation and status and prestige to existence. Now, embedded in that existence, we may still play games of power, but it will be conditioned that existence itself is the highest virtue.' In March, Johnson announced that he was 'building a religion' that would soon become history's 'fastest-growing ideology.' Don't Die or DD, 'means something different to everyone,' Johnson writes on DD's website , where people are invited to 'join or die.' Among its goals, that earth be 'a sustainable planet for those who choose life, and to secure the future of human existence with the rise of artificial intelligence.' Step 1: 'Identify the source of death,' meaning biological aging. Step 2: 'State your goal,' ideally to 'personally achieve age escape velocity where one year passes and I remain the same biological age.' According to Johnson, 5,000 people have so far committed to DD's 'Self Experimentation Study,' rigorously measuring their biomarkers, while 127 leaders have launched 'Don't Die' communities in their city. National Post
Edmonton Journal
2 hours ago
- Edmonton Journal
Why biohacker Bryan Johnson wants to sell his anti-aging business to build a new religion
Tech entrepreneur and longevity crusader is thinking of ditching his 'pain-in-the-ass' anti-aging nutrition and supplements company to focus on his 'Don't Die' movement, a community of likeminded biohackers 'united in defeating all causes of human and planetary death.' Article content In a lengthy interview with Wired, the 47-year-old California multimillionaire said he is 'so close' to shutting down or selling Blueprint, a wellness company devoted to 'maximally slowing' aging and reversing aging that's already occurred. Article content Article content Article content 'I've been talking to people about this. I don't need the money, and it's a pain-in-the-ass company,' Johnson told Wired's Katie Drummond. Article content Article content Johnson said Blueprint evolved from his own personal search for a clean, low-in-heavy-metals protein powder into a business venture that was just 'trying to do people a solid. The problem is now people see the business and give me less credibility on the philosophy side. I will not make that trade off … So yeah, I don't want it.' Article content Here's what to know about Johnson, Blueprint, his new religion and why he believes a crude AI copy of Bryan already exists. Article content Johnson, who told Wired he grew up poor ('My mom made my clothes') and remained so until he was 34, made a vast sum after selling his mobile payment processing platform to PayPal for a reported US$800 million, according to Fortune. Article content The self-described most measured human on the planet, Johnson takes 40 odd vitamins and supplements daily as well as hundreds of daily measurements of his heart, liver, lungs, kidneys and other body organs so that they may 'speak for themselves what they need to be in their ideal state.' Article content In 2023, Johnson, his then 17-year-old son and Johnson's father participated in a multi-generational plasma exchange. Johnson received plasma from a litre of blood siphoned from his son at a Texas spa in the hope his son's blood would make him younger. Article content The plasma swap apparently had a null effect: In January, Johnson posted on X that he was no longer injecting his son's blood and had 'upgraded' to another controversial plasma protocol. Article content Article content I am no longer injecting my son's blood. I've upgraded to something else: total plasma exchange. Steps: 1. Take out all blood from body 2. Separate plasma from blood 3. Replace plasma with 5% albumin & IVIG Here's my bag of plasma. Who wants it? 🧵 — Bryan Johnson (@bryan_johnson) January 28, 2025 Article content Johnson eats all the day's food before noon and sticks to a strict high-fibre, 'veggies and legumes,' no alcohol, no sugar diet that makes him feel sharper while avoiding 'post meal dead zones,' he's shared on X. Article content Last year, in a therapy dubbed 'Project Baby Face,' Johnson attempted to restore volume he's lost on his face from a calorie-reduced diet with fat injections in his temple, cheeks and chin. He didn't have enough of is own body fat so he used donor fat. It didn't go well: 'Immediately following the injections, my face began to blow up,' Johnson posted on Instagram. 'And then it got worse, and worse, and worse until I couldn't even see,' a severe allergic reaction. Article content What is Project Blueprint? Article content Johnson has explained how, In 2021, 'I endeavoured to figure out proximity to longevity escape velocity. How far away are we from one year of chronological time passing and one staying the same age biologically? I called this Project Blueprint.'
Cision Canada
2 hours ago
- Cision Canada
High‑Growth Oncology Market Projected For US$900bn in Revenue Despite Policy Headwinds
VANCOUVER, BC, July 23, 2025 /CNW/ -- USA News Group News Commentary – Cancer diagnoses are climbing fastest among younger women, sparking alarm that looming cuts to U.S. research budgets could stall progress in prevention and treatment. Adding to the strain, federal and state regulators are re‑examining mRNA vaccines, creating a cloud of uncertainty over future cancer‑funding priorities. Yet even amid this policy turbulence, a new wave of oncology innovators is lining up near‑term milestones that could reshape the landscape—led by Oncolytics Biotech Inc. (NASDAQ: ONCY) (TSX: ONC), Cue Biopharma, Inc. (NASDAQ: CUE), Verastem, Inc. (NASDAQ: VSTM), Allogene Therapeutics, Inc. (NASDAQ: ALLO), and Perspective Therapeutics, Inc. (NYSE-American: CATX). While regulatory red tape tightens, surging diagnoses of colorectal and other GI cancers in young people demand quicker innovation in oncology. Forecasts suggest worldwide cancer‑drug revenues could blow past US$900 billion by 2034. Within that, next‑gen therapies built on precision techniques are slated to reach US$175.2 billion, advancing at a brisk 7.35% annual clip. Oncolytics Biotech Inc. (NASDAQ: ONCY) (TSX: ONC) recently held a well‑attended key opinion leader (KOL) webinar where pancreatic and gastrointestinal cancer specialists dissected pelareorep's clinical history, its biomarker data package, and where the oncolytic virus could slot into evolving chemotherapy and immunotherapy regimens. "I want to thank our esteemed panel of oncologists for their meaningful contributions to our KOL event," said Jared Kelly, CEO of Oncolytics. "Their insights underscore a critical and timely message: pelareorep is a compelling immunotherapy platform that is well situated for combination strategies and a highly differentiated asset for pharma partners looking to expand or establish leadership in GI oncology. We are committed to moving forward aggressively toward registrational development while engaging with partners who share our vision of transforming outcomes in these difficult-to-treat cancers." The panel revisited survival gains in metastatic pancreatic ductal adenocarcinoma (mPDAC), reviewed translational evidence of "cold‑to‑hot" tumor conversion, and outlined next steps for a registration-enabling study. Management said feedback from the discussion would inform both upcoming trial designs and partnering talks now underway. The event came on the heels of Oncolytics having rolled out an expanded translational‑data review that tightened the scientific case for pelareorep, an intravenously delivered oncolytic reovirus. Renewed analyses from the GOBLET gastrointestinal cancer study and the AWARE‑1 breast cancer study confirmed that the virus replicates inside tumors, switches on interferon signaling in the immune system, and draws tumor‑infiltrating lymphocytes into the tumor microenvironment. "This robust data set, amassed from several studies in cancers that have historically resisted immunotherapeutic approaches, provides definitive validation of pelareorep's immune-mediated mechanism of action," said Dr. Thomas Heineman, Chief Medical Officer of Oncolytics. "We observed tumor biopsy-confirmed virus replication, immune cell activation, and the recruitment of cytotoxic T cells into the TME – all consistent with the durable responses observed in patients with metastatic PDAC and HR+/HER2- breast cancer who were treated with pelareorep." Investigators also recorded higher PD‑L1 expression and tracked newly expanded cytotoxic T‑cell clones in blood samples that matched those inside shrinking lesions—molecular fingerprints that point to stronger responses when pelareorep is combined with standard-of-care treatments and checkpoint inhibitors. "The collection of data here show that pelareorep works how a cancer immunotherapy should work," said Jared Kelly, CEO of Oncolytics. "Pelareorep is a versatile product candidate with strong platform potential to enhance immunological responses in multiple indications, including hard-to-treat cancers. Such compelling findings should be exciting to strategic partners focused on finding a platform immunotherapy in large indications with high unmet medical needs." Clinical outcomes already hint at real‑world benefit. In more than 100 first‑line mPDAC patients, pelareorep‑based regimens achieved a 21.9% two‑year overall‑survival rate versus a 9.2% historical benchmark. A separate single‑arm study that paired pelareorep with chemotherapy and a checkpoint blocker produced a 62% objective‑response rate—especially notable given that no checkpoint therapy is approved in this cancer today. In hormone‑receptor‑positive, HER2‑negative metastatic breast cancer (HR+/HER2‑ mBC), two randomized trials added more than ten months of median overall survival, while BRACELET‑1 nearly doubled median progression‑free survival to 12.1 months compared with 6.4 months for control patients. To steer these data toward value‑creating deals and late‑stage trials, the company strengthened its leadership earlier this year by appointing industry veteran Jared Kelly as CEO and naming Andrew Aromando Chief Business Officer. The duo previously helped guide Ambrx Biopharma into a US$2 billion sale to Johnson & Johnson, experience the board believes will support capital‑efficient development and strategic partnering for pelareorep. "Pelareorep's clinical data across multiple tumors is striking and represents the potential for a true backbone immunotherapy to address many in-need indications," said Kelly. "With a renewed focus and sharpened clinical development plan, we believe we will move pelareorep forward effectively and efficiently to a place where potential partners will see the value of a de-risked immunotherapy." As CBO, Aromando is now leading global business development and helping shape the company's corporate, clinical, and regulatory strategies. The leadership tandem is expected to prioritize partnering and expansion opportunities while preserving capital efficiency—a strategy well-suited for pelareorep's growing clinical profile. "I'm thrilled to join Oncolytics at such a pivotal moment in its evolution," said Aromando. "With promising data in difficult-to-treat cancers and a compelling body of clinical evidence in over 1,100 patients, I believe the Company is uniquely positioned to deliver meaningful value to patients and other stakeholders in the near term." Pelareorep currently holds FDA Fast Track designations in mPDAC (pancreatic cancer) and HR+/HER2‑ mBC (breast cancer), along with Orphan‑Drug status for pancreatic cancer in the United States and Europe. With mechanistic proof, survival data that outperform historical controls, and fresh validation from the recent KOL event, Oncolytics Biotech is aligning its clinical, regulatory, and business strategies to move pelareorep into registration‑enabling trials and position it as a backbone immunotherapy across solid tumors. In other recent industry developments and happenings in the market include: Cue Biopharma, Inc. (NASDAQ: CUE) recently reported that adding CUE‑101 to pembrolizumab produced a 50% overall response rate in patients with recurrent/metastatic HPV‑positive head and neck cancer who had a combined positive score (CPS) ≥ 1, including the same 50% response in those with low CPS 1–19. The regimen has now generated two complete responses, an 88% 12‑month overall survival rate, and a median overall survival of 32 months, handily outperforming historical pembrolizumab data. "The culmination of maturing data further support our conviction that CUE-101, representative of our approach with the CUE-100 series, demonstrates a potential breakthrough therapeutic approach for establishing a new standard of care," said Dan Passeri, CEO of Cue Biopharma. "With this maturing data, we are further emboldened in our conviction that our Immuno-STAT ® platform represents transformative potential for selectively modulating the patient's immune system." Verastem, Inc. (NASDAQ: VSTM) recently published Phase 2 RAMP 201 results in the Journal of Clinical Oncology, showing avutometinib + defactinib delivered a 31% confirmed overall response rate in recurrent low‑grade serous ovarian cancer, rising to 44% in KRAS‑mutant tumors. "The publication of the primary analysis of the RAMP 201 study in recurrent low-grade serous ovarian cancer in the Journal of Clinical Oncology reflects the meaningful clinical advancement demonstrated by the combination of avutometinib plus defactinib for patients with recurrent low-grade serous ovarian cancer," said John Hayslip, M.D., Chief Medical Officer at Verastem Oncology. "The findings supported the recent FDA approval of the combination in KRAS-mutated recurrent low-grade serous ovarian cancer and our ongoing global Phase 3 RAMP 301 trial of the combination in recurrent low-grade serous ovarian cancer with and without a KRAS mutation." Median progression‑free survival reached 12.9 months overall and 31.0 months in the KRAS‑mutant subgroup, with 82% of patients experiencing some tumor shrinkage regardless of mutation status. Back in June, Allogene Therapeutics, Inc. (NASDAQ: ALLO) presented updated Phase 1 TRAVERSE data showing a single dose of ALLO‑316 achieved a 31% confirmed response rate in heavily pre‑treated renal cell carcinoma patients whose tumors expressed CD70 in ≥ 50 % of cells. "ALLO-316 is showing clear evidence of targeted antitumor activity in patients who had failed most or all approved therapies for advanced or metastatic renal cell carcinoma," said Zachary Roberts, M.D., Ph.D., EVP, Research and Development and Chief Medical Officer at Allogene. "Our proprietary Dagger technology allows the use of a standard cyclophosphamide and fludarabine-based lymphodepletion regimen with a single dose of ALLO-316. Strong CAR T-cell kinetics and extensive infiltration of tumor tissue by CAR T cells are combining to generate deep and durable remissions. These are results that were previously considered out of reach for patients with advanced solid tumors." Four of five responders remain in remission—including one lasting more than 12 months—while safety remained manageable with no graft‑versus‑host disease observed. Investigators say the results highlight allogeneic CAR T's potential in solid tumors and justify continued expansion of the study. Perspective Therapeutics, Inc. (NYSE-American: CATX) recently began recruiting the third dose‑escalation cohort of its Phase 1/2a trial testing [²¹²Pb]VMT‑α‑NET in unresectable or metastatic somatostatin receptor 2 (SSTR2)‑positive neuroendocrine tumors, raising the fixed dose by 20% to 6 mCi. Earlier cohorts showed anti‑tumor activity with mostly low‑grade adverse events, prompting FDA alignment to explore whether the higher dose can further enhance efficacy. "We are excited to start exploring a higher dose level of VMT-α-NET after successfully completing an interaction with the FDA that was agreed prior to commencement of this trial," commented Markus Puhlmann, Chief Medical Officer of Perspective. "We are encouraged by the overall clinical profile observed at the second dose level of VMT-α-NET—including evidence of anti-tumor activity and primarily low-grade adverse events—and we believe it is important to assess whether a higher dose could further improve the therapeutic profile." The company plans additional safety and efficacy readouts, including dosimetry analyses, at scientific meetings in 2H 2025. DISCLAIMER: Nothing in this publication should be considered as personalized financial advice. We are not licensed under securities laws to address your particular financial situation. No communication by our employees to you should be deemed as personalized financial advice. Please consult a licensed financial advisor before making any investment decision. This is a paid advertisement and is neither an offer nor recommendation to buy or sell any security. We hold no investment licenses and are thus neither licensed nor qualified to provide investment advice. The content in this report or email is not provided to any individual with a view toward their individual circumstances. USA News Group is a wholly-owned subsidiary of Market IQ Media Group, Inc. ("MIQ"). MIQ has been paid a fee for Oncolytics Biotech Inc. advertising and digital media from the company directly. There may be 3rd parties who may have shares of Oncolytics Biotech Inc., and may liquidate their shares which could have a negative effect on the price of the stock. This compensation constitutes a conflict of interest as to our ability to remain objective in our communication regarding the profiled company. Because of this conflict, individuals are strongly encouraged to not use this publication as the basis for any investment decision. The owner/operator of MIQ own shares of Oncolytics Biotech Inc. which were purchased in the open market, and reserve the right to buy and sell, and will buy and sell shares of Oncolytics Biotech Inc. at any time without any further notice commencing immediately and ongoing. We also expect further compensation as an ongoing digital media effort to increase visibility for the company, no further notice will be given, but let this disclaimer serve as notice that all material, including this article, which is disseminated by MIQ has been approved by Oncolytics Biotech Inc.; this is a paid advertisement, we currently own shares of Oncolytics Biotech Inc. and will buy and sell shares of the company in the open market, or through private placements, and/or other investment vehicles. While all information is believed to be reliable, it is not guaranteed by us to be accurate. Individuals should assume that all information contained in our newsletter is not trustworthy unless verified by their own independent research. Also, because events and circumstances frequently do not occur as expected, there will likely be differences between the any predictions and actual results. Always consult a licensed investment professional before making any investment decision. Be extremely careful, investing in securities carries a high degree of risk; you may likely lose some or all of the investment.
