fda

from-birth-to-gene-edited-in-6-months:-custom-therapy-breaks-speed-limits

From birth to gene-edited in 6 months: Custom therapy breaks speed limits

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In the boy’s fourth month, researchers were meeting with the Food and Drug Administration to discuss regulatory approval for a clinical trial—a trial where KJ would be the only participant. They were also working with the institutional review board (IRB) at Children’s Hospital of Philadelphia to go over the clinical protocol, safety, and ethical aspects of the treatment. The researchers described the unprecedented speed of the oversight steps as being “through alternative procedures.”

In month five, they started toxicology testing in mice. In the mice, the experimental therapy corrected KJ’s mutation, replacing the errant A-T base pair with the correct G-C pair in the animals’ cells. The first dose provided a 42 percent whole-liver corrective rate in the animals. At the start of KJ’s sixth month, the researchers had results from safety testing in monkeys: Their customized base-editing therapy, delivered as mRNA via a lipid nanoparticle, did not produce any toxic effects in the monkeys.

A clinical-grade batch of the treatment was readied. In month seven, further testing of the treatment found acceptably low-levels of off-target genetic changes. The researchers submitted the FDA paperwork for approval of an “investigational new drug,” or IND, for KJ. The FDA approved it in a week. The researchers then started KJ on an immune-suppressing treatment to make sure his immune system wouldn’t react to the gene-editing therapy. Then, when KJ was still just 6 months old, he got a first low dose of his custom gene-editing therapy.

“Transformational”

After the treatment, he was able to start eating more protein, which would have otherwise caused his ammonia levels to skyrocket. But he couldn’t be weaned off of the drug treatment used to keep his ammonia levels down (nitrogen scavenging medication). With no safety concerns seen after the first dose, KJ has since gotten two more doses of the gene therapy and is now on reduced nitrogen scavenging medication. With more protein in his diet, he has moved from the 9th percentile in weight to 35th or 40th percentile. He’s now about 9 and a half months old, and his doctors are preparing to allow him to go home from the hospital for the first time. Though he will have to be closely monitored and may still at some point need a liver transplant, his family and doctors are celebrating the improvements so far.

From birth to gene-edited in 6 months: Custom therapy breaks speed limits Read More »

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Trump admin picks COVID critic to be top FDA vaccine regulator

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Oncologist Vinay Prasad, a divisive critic of COVID-19 responses, will be the next top vaccine regulator at the Food and Drug Administration, agency Commissioner Martin Makary announced on social media Tuesday.

Prasad will head the FDA’s Center for Biologics Evaluation and Research (CBER), which is in charge of approving and regulating vaccines and other biologics products, such as gene therapies and blood products.

“Dr. Prasad brings the kind of scientific rigor, independence, and transparency we need at CBER—a significant step forward,” Makary wrote on social media.

Prasad, a professor in the department of epidemiology and biostatistics at the University of California, San Francisco, is perhaps best known for his combative social media postings and criticism of the mainstream medical community. He gained notoriety amid the COVID-19 pandemic for assailing public health responses, such as masking and vaccine mandates.

In an October 2021 newsletter, titled “How Democracy Ends,” Prasad compared the country’s pandemic responses to the rise of Adolf Hitler’s Third Reich. The post led New York University bioethicist Arthur Caplan to rebuke Prasad, writing in The Cancer Letter that the comparison is “ludicrous, dangerous, and offensive,” before adding “imbecilic.”

Prasad has also criticized the FDA for approving COVID-19 booster vaccines. Last year, he accused his predecessor as the head of the CBER, Peter Marks, of being “either incompetent or corrupt” for allowing the approvals.

“Absurd”

More recently, Prasad has heaped praise on new FDA Commissioner Makary, while continuing to criticize Marks. In early March, Prasad called Makary “smart, thoughtful, and disciplined” and “exactly what we need at the FDA.” Later in the month, he continued to take shots at Marks, writing: “You could replace Peter Marks with a bobblehead doll that just stamps approval and you would have the same outcome at FDA with lower administrative fees. Maybe something DOGE should consider.”

Trump admin picks COVID critic to be top FDA vaccine regulator Read More »

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Seasonal COVID shots may no longer be possible under Trump admin

COVID-19, fda, health, makary, Trump, vaccines / /

Under President Trump, the Food and Drug Administration may no longer approve seasonal COVID-19 vaccines updated for the virus variants circulating that year, according to recent statements by Trump administration officials.

Since the acute phase of the pandemic, vaccine manufacturers have been subtly updating COVID-19 shots annually to precisely target the molecular signatures of the newest virus variants, which continually evolve to evade our immune responses. So far, the FDA has treated these tweaked vaccines the same way it treats seasonal flu shots, which have long been updated annually to match currently circulating strains of flu viruses.

The FDA does not consider seasonal flu shots brand-new vaccines. Rather, they’re just slightly altered versions of the approved vaccines. As such, the regulator does not require companies to conduct lengthy, expensive vaccine trials to prove that each slightly changed version is safe and effective. If they did, generating annual vaccines would be virtually impossible. Each year, from late February to early March, the FDA, the Centers for Disease Control and Prevention, and the World Health Organization direct flu shot makers on what tweaks they should make to shots for the upcoming flu season. That gives manufacturers just enough time to develop tweaks and start manufacturing massive supplies of doses in time for the start of the flu season.

So far, COVID-19 vaccines have been treated the exact same way, save for the fact that the vaccines that use mRNA technology do not need as much lead time for manufacturing. In recent years, the FDA decided on formulations for annual COVID shots around June, with doses rolled out in the fall alongside flu shots.

However, this process is now in question based on statements from Trump administration officials. The statements come amid a delay in a decision on whether to approve the COVID-19 vaccine made by Novavax, which uses a protein-based technology, not mRNA. The FDA was supposed to decide whether to grant the vaccine full approval by April 1. To this point, the vaccine has been used under an emergency use authorization by the agency.

Seasonal COVID shots may no longer be possible under Trump admin Read More »

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RFK Jr.‘s bloodbath at HHS: Blowback grows as losses become clearer

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Last week, Health Secretary and anti-vaccine advocate Robert F. Kennedy Jr. announced the Trump administration would hack off nearly a quarter of employees at the Department of Health and Human Services, which oversees critical agencies including the Food and Drug Administration (FDA), the Centers for Disease Control and Prevention (CDC), the National Institutes of Health (NIH), and the Centers for Medicare and Medicaid Services (CMS).

The downsizing includes pushing out about 10,000 full-time employees through early retirements, deferred resignations, and other efforts. Another 10,000 will be laid off in a brutal restructuring, bringing the total HHS workforce from 82,000 to 62,000.

“This will be a painful period,” Kennedy said in a video announcement last week. Early yesterday morning, the pain began.

It begins

At the FDA—which will lose 3,500 employees, about 19 percent of staff—some employees learned they were being laid off from security guards after their badges no longer worked when they showed up to their offices, according to Stat. At CMS—which will lose 300 employees, about 4 percent—laid-off employees were instructed to file any discrimination complaints they may have with Anita Pinder, identified as the director of CMS’s Office of Equal Opportunity and Civil Rights. However, Pinder died last year, The Washington Post noted.

At the NIH—which is set to lose 1,200 employees, about 6 percent—new director Jay Bhattacharya sent an email to staff saying he would implement new policies “humanely,” while calling the layoffs a “significant reduction.” Five NIH institute directors and at least two other senior leaders have been ousted, in addition to hundreds of lower-level employees. Bhattacharya wrote that the remaining staff will have to find new ways to carry out “key NIH administrative functions, including communications, legislative affairs, procurement, and human resources.”

At CDC—which will lose 2,400 employees, about 18 percent—the cuts slashed employees working in chronic disease prevention, sexually transmitted diseases, HIV, tuberculosis, global health, environmental health, occupational safety and health, maternal and child health, birth defects, violence prevention, health equity, communications, and science policy.

Some leaders and workers at the CDC and NIH were reportedly reassigned or offered transfers to work at the Indian Health Services (IHS), an HHS division that provides medical and health services to Native American tribes. The transfers, which could require employees to move to a remote branch, are seen as another way to force workers out.

RFK Jr.‘s bloodbath at HHS: Blowback grows as losses become clearer Read More »

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Judge orders Trump admin. to restore CDC and FDA webpages by midnight

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“Irrational removal”

In his opinion, Bates cited the declarations from Stephanie Liou, a physician who works with low-income immigrant families and an underserved high school in Chicago, and Reshma Ramachandran, a primary care provider who relies on CDC guidance on contraceptives and sexually transmitted diseases in her practice. Both are board members of Doctors for America.

Liou testified that the removal of resources from the CDC’s website hindered her response to a chlamydia outbreak at the high school where she worked. Ramachandran, meanwhile, testified that she was left scrambling to find alternative resources for patients during time-limited appointments. Doctors for America also provided declarations from other doctors (who were not members of Doctors for America) who spoke of being “severely impacted” by the sudden loss of CDC and FDA public resources.

With those examples, Bates agreed that the removal of the information caused the doctors “irreparable harm,” in legal terms.

“As these groups attest, the lost materials are more than ‘academic references’—they are vital for real-time clinical decision-making in hospitals, clinics and emergency departments across the country,” Bates wrote. “Without them, health care providers and researchers are left ‘without up-to-date recommendations on managing infectious diseases, public health threats, essential preventive care and chronic conditions.’ … Finally, it bears emphasizing who ultimately bears the harm of defendants’ actions: everyday Americans, and most acutely, underprivileged Americans, seeking healthcare.”

Bates further noted that it would be of “minimal burden” for the Trump administration to restore the data and information, much of which has been publicly available for many years.

In a press statement after the ruling, Doctors for America and Public Citizen celebrated the restoration.

“The judge’s order today is an important victory for doctors, patients, and the public health of the whole country,” Zach Shelley, a Public Citizen Litigation Group attorney and lead counsel on the case, said in the release. “This order puts a stop, at least temporarily, to the irrational removal of vital health information from public access.”

Judge orders Trump admin. to restore CDC and FDA webpages by midnight Read More »

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FDA approves first non-opioid pain medicine in more than 20 years

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The approval “is an important public health milestone in acute pain management,” Jacqueline Corrigan-Curay, J.D., M.D., acting director of the FDA’s Center for Drug Evaluation and Research, said in a statement. “A new non-opioid analgesic therapeutic class for acute pain offers an opportunity to mitigate certain risks associated with using an opioid for pain and provides patients with another treatment option.”

The company behind the drug, Vertex, said a 50 mg pill that works for 12 hours will have a wholesale cost of $15.50, making the daily cost $31 and the weekly cost $217. The cost is higher than cheap, generic opioids. But, a report from The Institute for Clinical and Economic Review in December estimated that suzetrigine would be “slightly cost-saving” relative to opioids if the price was set at $420 per week, given the drug’s ability to avert opioid addiction cases.

In a statement, Reshma Kewalramani, the CEO and President of Vertex, trumpeted the approval as a “historic milestone for the 80 million people in America who are prescribed a medicine for moderate-to-severe acute pain each year … [W]e have the opportunity to change the paradigm of acute pain management and establish a new standard of care.”

FDA approves first non-opioid pain medicine in more than 20 years Read More »

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Biofilms, unwashed hands: FDA found violations at McDonald’s ex-onion supplier

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Perhaps most concerning, FDA inspectors noted that employees were never seen washing their hands. Instead, they wore gloves, and if they touched dirty surfaces or items, they would simply put hand sanitizer on their dirty gloves and carry on. What they should have been doing was removing their dirty gloves, washing their hands, and getting clean gloves. However, the FDA inspectors never saw this happen, and managers confirmed that hand sanitizing gloves was common practice.

The inspectors also noted that the facility’s equipment was always wet. Employees applied sanitizing solutions on knives and other equipment used to dice and chop fresh produce. The sanitizing solution is meant to be air-dried before use, but Taylor Farms employees immediately used the equipment—still dripping with sanitizing solution—to cut RTE produce.

On one day of the inspection, FDA agents saw employees chop RTE lettuce with equipment that was wet with sanitizing solution at the maximum concentration, which was 200 ppm. In another instance, the inspectors saw an employee mix cleaning chemicals together to make a sanitizing solution, which the employee said was done “routinely.” When inspectors asked about the mixture, Taylor Farms “could not find the source of this recipe,” nor could they find the manufacturer label or other information stating the mixture was designed for use.

“Highest expectations”

In a statement to CBS, Taylor Farms said that it “immediately took steps to address” the problems found in the FDA’s inspection, which resulted in no “administrative or regulatory action” against the company.

“Taylor Farms is confident in our best-in-class food safety processes, and in turn, the quality and safety of our products,” the company said in the statement. “As is common following an inspection, FDA issued observations of conditions that could be improved at one of our facilities.” The company added that “no illnesses or public health threat has been linked to these observations” in the inspection.

McDonald’s, meanwhile, said it had stopped getting onions from the facility.

“We hold our suppliers to the highest expectations and standards of food safety. Prior to this inspection, and unrelated to its findings, McDonald’s stopped sourcing from Taylor Farms’ Colorado Springs facility,” McDonald’s said in a statement.

Biofilms, unwashed hands: FDA found violations at McDonald’s ex-onion supplier Read More »

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After decades, FDA finally moves to pull ineffective decongestant off shelves

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In a long-sought move, the Food and Drug Administration on Thursday formally began the process of abandoning oral doses of a common over-the-counter decongestant, which the agency concluded last year is not effective at relieving stuffy noses.

Specifically, the FDA issued a proposed order to remove oral phenylephrine from the list of drugs that drugmakers can include in over-the-counter products—also known as the OTC monograph. Once removed, drug makers will no longer be able to include phenylephrine in products for the temporary relief nasal congestion.

“It is the FDA’s role to ensure that drugs are safe and effective,” Patrizia Cavazzoni, director of the FDA’s Center for Drug Evaluation and Research, said in a statement. “Based on our review of available data and consistent with the advice of the advisory committee, we are taking this next step in the process to propose removing oral phenylephrine because it is not effective as a nasal decongestant.”

For now, the order is just a proposal. The FDA will open up a public comment period, and if no comments can sway the FDA’s previous conclusion that the drug is useless, the agency will make the order final. Drugmakers will get a grace period to reformulate their products.

Reviewed reviews

The slow-moving abandonment of phenylephrine is years in the making. The decongestant was originally approved by the FDA back in 1976, but it came to prominence after 2006. That was the year when the “Combat Methamphetamine Epidemic Act of 2005” came into effect, and pseudoephedrine—the main component of Sudafed—moved behind the pharmacy counter to keep it from being used to make methamphetamine. With pseudoephedrine out of easy reach at drugstores, phenylephrine became the leading over-the-counter decongestant. And researchers had questions.

In 2007, an FDA panel reevaluated the drug, which allegedly works by shrinking blood vessels in the nasal passage, opening up the airway. While the panel upheld the drug’s approval, it concluded that more studies were needed for a full assessment. After that, three large, carefully designed studies were conducted—two by Merck for the treatment of seasonal allergies and one by Johnson & Johnson for the treatment of the common cold. All three found no significant difference between phenylephrine and a placebo.

After decades, FDA finally moves to pull ineffective decongestant off shelves Read More »

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“Havard”-trained spa owner injected clients with bogus Botox, prosecutors say

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Mounting evidence

Multiple clients and employees told investigators that Fadanelli also said she is a registered nurse, which is false. Though she is a registered aesthetician, aestheticians are not permitted to administer injections or prescription drugs.

Investigators set up an undercover operation where an agent went in for a consultation, and Fadanelli provided a quote for a $450 Botox treatment. Investigators also obtained videos and images of Fadanelli performing injections. And the evidence points to those injections being counterfeit, prosecutors allege. Sales records from the spa indicate that Fadanelli performed 1,631 “Botox” injections, 95 “Sculptra” injections, and 990 injections of unspecified “filler,” all totaling over $933,000. But sales records from the manufacturers of the brand name drugs failed to turn up any record of Fadanelli or anyone else from her spa ever purchasing legitimate versions of the drugs.

Despite the mounting evidence against her, Fadanelli reportedly stuck to her story, denying that she ever told anyone she was a nurse and denying ever administering any injections. “When agents asked Fadanelli if she would like to retract or modify that claim if she knew there was evidence showing that she was in fact administering such products, she reiterated that she does not administer injections.”

Ars has reached to Fadanelli’s spa for comment and will update this story if we get a response. According to the affidavit, clients who received the allegedly bogus injections complained of bumps, tingling, and poor appearances, but no infections or other adverse health outcomes.

In a press release announcing her arrest, Acting United States Attorney for Massachusetts Joshua Levy said: “For years, Ms. Fadanelli allegedly put unsuspecting patients at risk by representing herself to be a nurse and then administering thousands of illegal, counterfeit injections. … The type of deception alleged here is illegal, reckless, and potentially life-threatening.”

For a charge of illegal importation, Fadanelli faces up to 20 years in prison and a $250,000 fine. For each of two charges of knowingly selling or dispensing a counterfeit drug or counterfeit device, she faces up to 10 years in prison and a fine of $250,000.

“Havard”-trained spa owner injected clients with bogus Botox, prosecutors say Read More »

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Drugmaker shut down after black schmutz found in injectable weight-loss drug

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It’s unclear how widely the pharmacy’s drugs were distributed. Fullerton Wellness could not be reached for comment.

Bigger battles

This is just the latest warning on weight-loss drugs from the FDA, which has repeatedly cautioned about quality and safety problems related to compounded versions of the drugs. The compounded drugs are intended to be essentially copycat versions of the blockbuster brand-name drugs. Compounding pharmacies can make copycat versions only as long as the drugs are in short supply, acting as a stopgap for patient access. But, with the popularity of the drugs and the high prices of the brand name versions, compounded formulations have become seen as affordable alternatives for many patients.

The situation has become a legal quagmire, with less-than-scrupulous compounding facilities drawing the ire of the FDA, and the big pharmaceutical companies fighting with their compounding competition. Eli Lilly, maker of Zepbound and Mounjaro (tirzepatide), and Novo Nordisk, maker of Wegovy and Ozempic (semaglutide), have both sued multiple compounding pharmacies over copycat versions of their lucrative drugs, which they claim are unsafe and fraudulent.

Meanwhile, in October, a trade organization for large-scale compounding pharmacies sued the FDA after the regulator removed tirzepatide from the drug shortage list, a move that blocks compounders from making copycat versions of the drug. But, the FDA quickly backpedaled in court, saying it would reconsider the removal and would allow compounders to keep producing off-brand versions in the meantime.

Also in October, Novo Nordisk asked the FDA to stop letting compounders make copycat versions of semaglutide, arguing that the drug is too complex for compounders to make and poses too many safety risks to patients. In response, the trade organization for compounders, the Outsourcing Facilities Association, submitted a letter to the FDA asking it to require Novo Nordisk to provide an economic impact statement to assess the cost and price increases that could occur if semaglutide were no longer available through compounding pharmacies.

Drugmaker shut down after black schmutz found in injectable weight-loss drug Read More »

guy-makes-“dodgy-e-bike”-from-130-used-vapes-to-make-point-about-e-waste

Guy makes “dodgy e-bike” from 130 used vapes to make point about e-waste

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Disposable vapes are indefensible. Many, or maybe most, of them contain rechargeable lithium-ion batteries, but manufacturers prefer to sell new ones. More than 260 million vape batteries are estimated to enter the trash stream every year in the UK alone. Vapers and vape makers are simply leaving an e-waste epidemic to the planet’s future residents to sort out.

To make a point about how wasteful this practice is—and to also make a pretty rad project and video—Chris Doel took 130 disposable vape batteries (the bigger “3,500 puff” types with model 20400 cells) found littered at a music festival and converted them into a 48-volt, 1,500-watt e-bike battery, one that powered an e-bike with almost no pedaling more than 20 miles. You can see the whole build and watch Doel zoom along trails on his YouTube video.

A pile of empty aluminum vape shells, and the juice and batteries that came out of them, on Chris Doel’s workstation. A pile of empty aluminum

To be clear: Do not do this. Do not put disposable vape cartridges in a vise clamp to “pop out” their components. Do not desolder them from vape cartridges that have a surprising amount of concentration still in them. Do not wire them together using a balance board, group them using 3D-printed cell holders, and then wire them in series. Heck, do not put that much power into a rear hub on a standard bike frame, at least more than once. Doel has a fire extinguisher present and visible on his workbench, and he shows you what happens when two of the wrong batteries happen to make momentary contact—smoke, coughing, and strong warnings.

And yet, when you see Doel get 33 kilometers (about 20.5 miles) on his vape-powered ride, almost entirely without pedaling, hitting 32 miles per hour once, the point is made. We are tossing out a lot of battery materials that could be doing a lot of other things. Doel estimates his “dodgy bike” cost about $60 in filament and materials to piece together. Most of the cells are rated for a minimum of 300 cycles, which is both not a lot, but also more than some bikes and scooters actually take on.

Guy makes “dodgy e-bike” from 130 used vapes to make point about e-waste Read More »

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Shady drugmaker used code words to sell knockoff weight-loss drug: lawsuit

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Starts with a T

Pivotal Peptides—which is not a licensed pharmacy or dispensary—did not respond to the letter. Instead, its website was modified to indicate that it was “down for maintenance,” and the company instructed customers to email directly. About 10 days later, Pivotal Peptides’ registered agent, Elizabeth Gately, then sent an email (which Lilly obtained) instructing customers to place tirzepatide orders using coded language.

“Good News,” the email read, “Pivotal Peptides … is still in business!”

“If a favorite product (starting with T) was your go-to, that name can’t be used in any correspondence with me or listed on my price sheet anymore,” Gately allegedly wrote. “Therefore, I need another identifier and decided (for now) to call this peptide ’11mg.'”

Gately went on to say that the codenamed product “is Pivotal Peptide’s [sic] bestseller,” and “it is the only T size available from PP right now except by special order.” The letter ended with: “Remember to order ’11 mg’ with the latest price to identify the product you want, if applicable, and no longer use T in our communication.”

Pivotal Peptides did not respond to Ars’ request for comment.

In a statement emailed to Ars, a Lilly spokesperson said Pivotal Peptides and the other companies Lilly is suing are engaging in “conduct that poses serious risks to patient safety.” In the lawsuit, Lilly notes that even children could be ordering this DIY, research-grade drug.

“No one should ever be allowed to sell these untested, non-human grade or manipulated drugs to American consumers,” the statement continued.

Lilly’s lawsuits come amid a legal storm over compounded versions of the tirzepatide, which can be legally made by licensed pharmacies as long as tirzepatide is in shortage. On October 2, the Food and Drug Administration announced that the shortage had ended but then decided to reconsider the decision after being sued by compounding pharmacies.

On several occasions, the FDA has warned of safety concerns related to compounded versions of GLP-1 weight-loss drugs.

Shady drugmaker used code words to sell knockoff weight-loss drug: lawsuit Read More »