Tag: fda

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 reported earlier in the year, had both received Elevidys, the only gene therapy approved by the FDA for the treatment of a rare form of muscular dystrophy called Duchenne muscular dystrophy (DMD), Sarepta officials said in a call on June 16.

The boys were both non-ambulatory and both died within 90 days of treatment, according to Sarepta.

The 15-year-old died from acute liver failure, Sarepta said in a statement on Sunday. Acute liver injury is listed as a side effect of the product.

Sarepta said it was investigating the cases independently and in relation to each other to identify any common risk factors. The company suspended its Elevidys sales forecast for this year and said it would provide an update with its second-quarter results.

Sarepta officials said the company is pausing shipments of Elevidys for non-ambulatory patients and halting a clinical trial while it develops an “enhanced immunosuppressive regimen,” utilizing the sirolimus drug. They said there is no timeline right now for resuming dosing for the population and that they would try to speak soon with FDA officials about the situation.

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Top FDA Official Admits She Refused the Covid-19 Vaccine While Pregnant

One of the most powerful figures at the US Food and Drug Administration (FDA) has admitted she refused the Covid-19 mRNA vaccine while pregnant—even as her agency promoted it as “safe and effective” for all pregnant women.

Dr Sara Brenner’s explosive disclosure, made on 15 May 2025 at the MAHA Institute Round Table in Washington, DC, is as revealing as it is troubling.

A preventive medicine physician, Brenner has worked at the FDA since 2019. As the FDA’s Principal Deputy Commissioner—and briefly its Acting Commissioner—Brenner was at the centre of decision-making.

Prior to that, she was Chief Medical Officer for diagnostics and was detailed to the White House to support the Biden administration’s Covid-19 response. She didn’t just participate in the pandemic response, she helped shape it from within.

“Knowing what I knew—not only about nanotechnology, about medicine, about the medical countermeasures—but also having a very strong and firm grounding in bioethics…there were many things that were not right,” she told the audience.

That someone with her seniority and access to internal data privately rejected the vaccine, while her agency promoted it to millions of pregnant women, presents a profound ethical dilemma.

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Smells Like Bureaucratic Overreach: FDA Greenlights Lab-Grown Salmon

In a move that reeks of bureaucratic overreach and questionable priorities, the Food and Drug Administration (FDA) has rubber-stamped Wildtype Inc.’s lab-grown salmon, the first of its kind approved for human consumption in the U.S.

Derived from mesenchymal lineage cells and mixed with plant-based goo to mimic sushi-grade saku cuts, this franken-fish is being hailed as a “sustainable” solution to overfishing. 

Meanwhile, HHS Secretary Robert F. Kennedy Jr. is shaking up the public health establishment, firing all 17 members of the CDC’s Advisory Committee on Immunization Practices (ACIP) in a bid to root out conflicts of interest and restore trust in vaccine science. 

The contrast couldn’t be starker: one agency pushes untested biotech food on the public, while another tries to clean house of industry-aligned insiders. Welcome to the brave new world of “Gold Standards.”

Wildtype’s cultured salmon, approved in June 2025 under FDA consultation CCC 000005, is grown in bioreactors over four to six weeks, sidestepping nature’s blueprint for a lab-bred alternative. 

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Texas Governor Signs Bill To Create Ibogaine Research Consortium, Aiming To Develop FDA-Approved Psychedelic Drug

Texas Gov. Greg Abbott (R) has signed into law a bill to create a state-backed research consortium to conduct clinical trials on ibogaine as a possible treatment for substance use disorders and other mental health conditions. The ultimate goal of the project is to develop the psychedelic into a prescription drug with U.S. Food and Drug Administration (FDA) approval, with the state retaining a portion of the profit.

“Texas is now leading the way in the United States for the evaluation of ibogaine as a potential medication that can help improve the lives of so many Americans,” the governor said at a signing event on Wednesday. He called the psychedelic “a therapy that has shown great promise in treating” conditions such as depression, PTSD and opioid use disorder.

“I’m about to sign a law that will lead to an FDA-approved drug development clinical trial that will seek approval of ibogaine as a medication for the treatment of opioid use disorder and other behavioral health conditions, especially those suffered by our veterans,” he added. “Texas will invest $50 million to support this research, and these funds can be matched by grants and private investments.”

Under the new law, approved by the state legislature earlier this month, Texas will retain a commercial interest in “all intellectual property that may be generated over the course of the drug development clinical trials,” the legislation says, with an aim of making Texas a hub for “ibogaine-related biomedical research, development, treatment, manufacturing, and distribution.”

A quarter of revenue taken in by the state from any resulting intellectual property would fund veterans programs.

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FDA Greenlights Merck’s RSV Shot for Newborns — Clinical Trials Showed 11.71% Rate of Serious Adverse Events, Including Death

The U.S. Food and Drug Administration (FDA) on Monday approved Merck’s new monoclonal antibody shot, which the company said is designed to protect newborns and infants against respiratory syncytial virus (RSV).

The antibody shot, clesrovimab, will be marketed as Enflonsia and launched this coming RSV season to challenge Sanofi and AstraZeneca’s blockbuster Beyfortus shot.

Beyfortus, first approved in 2023, generated $1.8 billion in sales last year, FiercePharma reported.

PR Newswire projected that the global RSV vaccine and antibody market would reach $13.59 billion by 2030.

Merck said its monoclonal antibody has about the same efficacy rate as Beyfortus. In clinical trials, the shot reduced RSV-related hospitalization by 84.3% compared to placebo, and the incidence of RSV that needed to be medically attended by 60.5% versus placebo, through age 5 months in the clinical trials.

The company also said in a press release that the drug’s safety profile was “generally comparable” to placebo. The most common adverse reactions were injection-site erythema or swelling, and a rash 14 days after the dose, all of which were more common in the vaccine group.

However, the clinical trial results posted on the government website ClinicalTrials.gov included other serious neurological adverse reactions that Dr. Peter Selley, a U.K. general practitioner who has closely followed the development of the drugs for RSV, called “concerning.”

These included febrile convulsions, seizures, facial paralysis and brain injury at about three times the rate in the vaccine group (25 of 2,409) as the placebo group (4 of 1,202).

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FDA Approved Hundreds of Drugs With No Evidence They Work

The U.S. Food and Drug Administration (FDA) has approved hundreds of drugs over the last several decades with little to no evidence that they work, according to a new investigation by The Lever.

Many of the drugs are permitted to stay on the market, despite ample evidence that they don’t work and that they cause serious and irreparable harm.

The Lever’s two-year investigation into the FDA-approved drugs analyzed government reports, internal FDA documents, investigators’ notes, congressional testimony, court records and more than 100 interviews with researchers, federal officials and patients.

The investigation found that from 2013 through 2022, 73% of drugs approved by the FDA didn’t meet the agency’s four foundational standards required to show the drugs work as expected. Fifty-five of the approved drugs met only one of those four standards, and 39 met none of them.

More than half of drug approvals were based on preliminary data, which meant the pharmaceutical companies didn’t submit evidence that patients had fewer symptoms, showed improvement or had their lives extended.

The approval rate of such drugs has accelerated over the last decade, according to the U.S. Department of Health and Human Services Office of Inspector General.

Cancer treatments, in particular, raised serious red flags. Only 2.4% of the 123 cancer drugs met all of the criteria, and 29 met none. The FDA approved 81% of cancer drugs based on preliminary data.

The report did not evaluate any vaccines.

The Lever wrote:

“These statistics come after billions of dollars and years of lobbying by the pharmaceutical industry and patient advocacy groups pressuring Congress to loosen the FDA’s scientific standards.

“The resulting seismic shift from proving drugs work before they are approved to showing they work only after approval — if ever — has been quietly accomplished with virtually no awareness by doctors or the public.

“Insurers and taxpayers effectively pay for research after drugs hit the market as pharmaceutical companies reap the profits. Patients serve as the unwitting guinea pigs — with very real consequences.”

The outlet also reported that an estimated 128,000 people are killed each year by side effects from prescription drugs prescribed as indicated.

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Liver Injuries Reportedly on the Rise: Common, FDA-Approved Meds Linked to Alarming Trend

As more people find themselves using more vitamins, supplements, and everyday medications, more related health issues keep cropping up.

And a recent spate of reports highlights a particular danger for one part of your body: the liver.

“As cases of drug-induced liver injury (DILI) are on the rise, experts are warning of the hidden dangers associated with some common medications and supplements,” Fox News reported Monday.

The outlet added, “Statistics show that DILI, also known as toxic hepatitis or hepatotoxicity — which is known to be a significant cause of acute liver failure — has been growing in Western countries since the 1960s.”

“Even medications that have been tested for safety and approved by the U.S. Food and Drug Administration (FDA) can potentially cause liver injury in rare cases,” Fox reported.

Just days earlier, on May 27, NBC News had posted a similarly alarming report regarding these health ramifications.

The outlet spoke to Robert Grafton, a former medical technologist who noticed signs of liver failure in himself and largely traced it back to various supplements and vitamins he had been taking.

“I stopped taking everything, thank goodness,” Grafton said. “If I hadn’t known, if I was not in the medical field, I might have thought, ‘Oh, I think I’m getting sick. I need to take some more of these supplements to help me feel better.’”

Despite stopping, symptoms worsened and test results pointed toward devastating liver failure.

It turned out to be a case of DILI.

“It is estimated that between 13.9 and 19.1 cases of DILI occur for every 100,000 people, according to recent research published in the journal Toxicology Reports,” Fox noted.

The triggers of DILI can vary, from the vitamins and supplements that Grafton had been taking, mentioned above, to aspirin, the common over-the-counter painkiller.

Those with health conditions are more susceptible to DILI, but as Grafton — someone who began taking his health seriously after having children — shows, it can hit very healthy people, too.

Fox News medical analyst Dr. Marc Siegel told his network that the unregulated nature of supplements could betray some of their purported health benefits.

“The biggest problem with herbal supplements is that the amount you are taking of active chemicals isn’t strictly regulated, so you don’t know exactly what you are getting,” he said. “And since several of the supplements are metabolized through the liver, there is now an increasing incidence of liver toxicity in users.”

According to the National Institutes of Health, “nearly all classes of medications” can cause DILI.

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An Evidence-Based Approach to Covid-19 Vaccination??

‘An Evidence-Based Approach to Covid-19 Vaccination’ is the misleading title of an extraordinary publication by Vinay Prasad and Marty Makary in the New England Journal of Medicine, 20th May 2025. It describes the FDA’s new approach, but if you were hoping for ‘Evidence-Based’ you’ll be sorely disappointed. One could argue that this is a start, in that it does involve withdrawing FDA recommendations for regular boosters for ‘healthy 6 months- to 64 year-olds’ but the state of ‘health’ in the US is so poor that apparently two thirds of Americans are still eligible for the shots. (‘Underlying medical conditions’ include pregnancy, obesity, physical inactivity and smoking, current and former),

The article goes on to describe a requirement for gold standard randomised controlled trials for further vaccines for a healthy population of say 50-64 years with a saline placebo and a 6 month follow-up but again using an end-point related to covid alone rather than all-cause mortality or morbidity. For over-65s or those with comorbidities, only evidence of antibody production is required.

Then on 27th May, a week after the NEJM article, Robert Kennedy, flanked by Jay Battacharia and Marty Makary, announced the changes to the CDC schedule, stating that the vaccines would be withdrawn for healthy under 65s and healthy pregnant women. The statement was somewhat underwhelming, especially Jay Battacharya’s rather wooden performance – another example of the 3-word or 3-phrase mantra: “That ends today. It’s common sense and it’s good science”.

Some cynics were quick to point out that the CDC guidance had not changed, but by 29th May the website was updated though not in quite the way I had anticipated from RFK’s words. The covid vaccine for children now states ‘See notes’ and then outlines a system of ‘shared clinical decision-making’ as follows: “Shared clinical decision-making vaccinations are individually based and informed by a decision process between the health care provider and the patient or parent/guardian. Where the parent presents with a desire for their child to be vaccinated, children 6 months and older may receive COVID-19 vaccination, informed by the clinical judgment of a healthcare provider and personal preference and circumstances.” Only for children with moderate or severe immunocompromise does the CDC actively advise a booster. So for children this actually goes further than the Prasad and Makary article, leaving only immunocompromise as a co-morbidity for recommended covid boosters in under 18s, in line with the current UK guidelines. The Children’s Health Defence have tried to explain the very opaque guidelines – one plus would be that paediatricians would no longer be obliged to recommend the vaccines – at present they risk losing their tenure if they speak out against the jabs. But for adults, there appears to be no change with covid vaccines still recommended as routine including during pregnancy.

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FDA Launches New AI Tool

The Food and Drug Administration on June 2 launched a new artificial intelligence (AI) tool.

FDA officials said Elsa, the tool, will help employees “work more efficiently.”

The agency is utilizing Elsa to speed up clinical protocol reviews and scientific evaluations, as well as to identify targets for inspections.

FDA officials described Elsa as a “large language model–powered AI tool designed to assist with reading, writing, and summarizing.” They said it can summarize adverse events to help with safety profile assessments, compare labels faster than humans, and generate code to help develop databases.

“Today marks the dawn of the AI era at the FDA with the release of Elsa, AI is no longer a distant promise but a dynamic force enhancing and optimizing the performance and potential of every employee,” FDA Chief AI Officer Jeremy Walsh said in a statement.

AI refers to computer systems that perform complex tasks typically performed by humans.

Dr. Marty Makary, the FDA’s commissioner, said in May that the FDA would immediately start using AI and fully integrate it by the end of June.

“Following a very successful pilot program with FDA’s scientific reviewers, I set an aggressive timeline to scale AI agency-wide by June 30,” Makary said on Monday.

“Today’s rollout of Elsa is ahead of schedule and under budget, thanks to the collaboration of our in-house experts across the centers.”

Health Secretary Robert F. Kennedy Jr., who leads the Department of Health and Human Services (HHS), the FDA’s parent agency, cheered the development, calling it “a revolution in public health” in a post on social media platform X.

The FDA recently fired thousands of employees. HHS officials had said they would cut about 3,500 full-time workers but ended up terminating about 2,500 workers, according to a Senate Democrat report.

Makary told a congressional panel during a recent appearance that no scientific reviewers were fired, although some research scientists were among those terminated.

President Donald Trump has promoted the use of AI, saying in one order that “with the right Government policies, we can solidify our position as the global leader in AI and secure a brighter future for all Americans.”

The first report from Trump’s Make America Healthy Again (MAHA) Commission, headed by Kennedy, contained markers of AI, including nonexistent studies and multiple instances of “oaicite,” which developers say is inserted by OpenAI’s ChatGPT AI tool.

“The pattern is consistent with other cases we’ve seen of using generative AI to create citations,” Dr. Ivan Oransky, co-founder of Retraction Watch and executive director of The Center for Scientific Integrity, told The Epoch Times in an email.

“The fact that these fake citations made it into the report are evidence that no one checked the report for rigor. That should concern anyone who reads it or considers relying on it.”

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FDA approves Moderna’s new lower-dose COVID-19 vaccine

The U.S. approved a new COVID-19 vaccine made by Moderna late Friday but with limits on who can use it — not a replacement for the company’s existing shot, but a second option.

The new vaccine, mNexspike, is a step toward next-generation coronavirus vaccines. It’s made in a way that allows for a lower dose — a fifth of the dose of its current COVID-19 vaccine, Spikevax — by refining its immune target.

The approval “adds an important new tool to help protect people at high risk of severe disease from COVID-19,” Stephane Bancel, Moderna’s CEO, said in a statement Saturday.

The Food and Drug Administration approved the new vaccine for use in all adults 65 and older, and for people age 12 to 64 who have a least one health condition that puts them at increased risk from the coronavirus.

That’s the same limit that the FDA set in licensing another COVID-19 vaccine option from competitor Novavax.

Those restrictions are a departure from how the U.S. has handled COVID-19 vaccines until now, reflecting skepticism about vaccines from Health Secretary Robert F. Kennedy Jr. and other Trump officials.

Moderna’s existing vaccine doesn’t face those limits and has long been used for anyone ages 6 months and older. The company said it expected to offer both options this fall.

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