Tag Archives: FDA

Families Fight for FDA Approval of Life-Saving Drug for Children

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Like many children affected by Hunter syndrome, Cole is missing a crucial enzyme required to break down specific molecules. As these toxins accumulate, they wreak havoc on vital organs, particularly the heart and brain, often resulting in dementia-like symptoms. This genetic disorder, also referred to as mucopolysaccharidosis type II (MPS II), impacts around 500 individuals in the United States, predominantly boys.

Experts believe the recently approved drug by Denali Therapeutics, an intravenous enzyme replacement therapy, could revolutionize treatment options, especially since current standard care only addresses the physical manifestations of the disease. Denali’s innovative therapy also aims to combat cognitive decline.

While new medications can’t reverse existing regression, they hold potential to extend a child’s life and stave off numerous symptoms when administered early.

Dr. Joseph Müntzer is an expert in Hunter syndrome and related rare diseases.
Alison M. Donnelly of NBC News

“If we can treat very young children before irreparable damage occurs, the possibilities are nearly limitless,” says Dr. Joseph Muenzer of the Muenzer MPS Research and Treatment Center at the University of North Carolina at Chapel Hill, who specializes in Hunter syndrome and other rare mucopolysaccharide disorders.

“I can’t predict how they will fare in the future, but it will certainly be different,” he remarked.

Before regression began, Cole learned to read and spoke in complete sentences. Gradually, his speech deteriorated, leaving him only able to say “Mommy” before losing all ability to communicate. Despite being a teenager now, he is developmentally similar to a 3-year-old.

The FDA’s approval of Denali’s drug was a significant relief for families with children suffering from Hunter syndrome and for the broader rare disease community. Recently, the FDA faced criticism for denying several promising treatments for rare conditions, prompting advocacy groups to stage protests and advocate for more supportive policies.

In an email to NBC News, the FDA noted that the number of drug approvals and denials under its current administration aligns with historical trends from the past decade. FDA Commissioner Dr. Marty McCurry hailed Denali’s approval as “a milestone day for children combating Hunter syndrome and their families,” pledging continued efforts to expedite treatments for rare diseases.

Many healthcare professionals, including Munzer, who led the Denali trial, are hopeful that advancements in treatments will continue.

“These are devastating diseases,” Munzer emphasized. “Their rarity should not lead to neglect.”

Denali’s drug, known as Avraya, marks the first FDA-approved treatment for Hunter syndrome in the United States in two decades and is the first to penetrate the blood-brain barrier, targeting the neurological complications associated with the disease.

Experts like Müntzer assert that Avraya could significantly extend life expectancy, based on encouraging human trial data. A clinical trial illustrated significant reductions in important disease biomarkers in the cerebrospinal fluid after just 24 weeks, with 93% of pediatric participants achieving levels akin to those without Hunter syndrome.

For countless families dealing with this condition, the approval of this drug signifies more than a new treatment; it acknowledges the inherent value of every child.

Laurent Jaskalski, a 6-year-old from New Berlin, Wisconsin, was diagnosed with Hunter syndrome at age four. He’s always struggled with communication, but his mother, Kylie Jaskalski, fears that his speech difficulties may lead others to underestimate his capabilities. This is a painful misconception about a joyful child who loves to cuddle and eagerly anticipates going to school each day.

“He brings immense joy,” Jaskalski said. “He spreads happiness and peace to everyone he meets.”

Six-year-old Roland Jaskalski, also known as Rory, diagnosed with Hunter syndrome at age 4.
Provided by Kylie Jaskalski

Initially, Jaskalski felt devastated when Laurent was diagnosed, but she later found it even more heart-wrenching to witness her son’s deterioration.

If insurance permits access to Denali’s drug for Laurent, “we might not have to remain passive,” she expressed.

Currently, Laurent receives weekly infusions of Elaplus (FDA-approved in 2006), which is the prevailing standard treatment. While this IV therapy isn’t flawless, it has stabilized his physical decline. Recently, Jaskalski has observed weakness and discomfort on Laurent’s left side, making walking challenging. He also suffers from mild hearing loss.

Recent advancements in newborn screening for Hunter syndrome across states like California to Rhode Island indicates a promising future, as early detection allows for timely intervention with new drugs, potentially mitigating cognitive decline.

Children with Hunter syndrome often face severe health challenges and a life expectancy ranging from 10 to 20 years. Those with the non-neurological variant may reach adulthood but still endure progressive physical complications, primarily affecting their airways and heart.

Three-year-old Kashton Estes refers to his weekly infusion of Elaplus, the standard treatment for Hunter syndrome, as his “Spider-Man juice.”
Provided by Christina Caldwell

The introduction of new treatments is especially hopeful for families with children across the spectrum of Hunter syndrome. Christina Caldwell’s 3-year-old grandson, Kashton Estes, who has the non-cognitive variant, undergoes IV infusions of Elaplus, which he affectionately calls “Spider-Man juice.” Thanks to this treatment, he enjoys running, jumping, and riding his bike.

Caldwell is eager for Kashton to be treated with Denali’s new drug as two of his cousins also have Hunter syndrome, and the family wishes to prevent future health complications for all three children.

“We’re not asking for much. Just provide us with the medication to keep our children alive,” she stated.

Regarding insurance coverage, Denali Therapeutics stated it has engaged in “constructive” dialogues with payers and is prioritizing swift access to Avraya for families dealing with Hunter syndrome. Currently, the drug is approved only for pediatric patients, but the company is working on expanding its clinical data to include young adults. Denali also aims to leverage its blood-brain barrier technology for various other diseases, including neurodegenerative disorders.

Ms. Stevens, the mother of 15-year-old Cole, eagerly anticipates administering the new medication to her son. She has devoted her life to aiding not just Cole but others in similar situations. In 2022, she took on the role of executive director at Munzer’s MPS Research and Treatment Center at UNC-Chapel Hill.

Upon learning last week about the FDA’s early approval for the new drug, Stevens rushed through the treatment center, sharing the news with patients and staff. There were tears of joy and hugs all around.

Cole Stevens Mausolf prepares for his weekly Elaplus infusion.
Alison M. Donnelly of NBC News

Stevens understands that Denali’s treatment cannot reverse Cole’s previous regression, meaning he may not attend university or secure a job. However, she remains profoundly grateful.

“My hope is for him to maintain stability,” Stevens expressed. “Even with a progressive disease, achieving stability is a victory.”

Source: www.nbcnews.com

FDA Proposes Ban on BHA Additive in Processed Foods Like Meat and Bread

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The Food and Drug Administration (FDA) announced on Tuesday that it is taking steps toward potentially banning BHA, a food additive used in various processed foods, including meats and breads.

Butylated hydroxyanisole (BHA) has been a part of our food supply for decades. The FDA first designated this chemical as “generally recognized as safe” in 1958 and approved it as a food additive in 1961. BHA is primarily used to prevent fats and oils from spoiling and can be found in products like frozen foods, breakfast cereals, cookies, ice cream, and certain meat items.

The FDA has stated that it will initiate a new safety review of BHA, addressing long-standing concerns regarding its potential carcinogenic effects in humans.

In the 1990s, the National Toxicology Program identified BHA as “reasonably expected to be a human carcinogen” based on animal studies. Moreover, it is recognized as a known carcinogen under California’s Proposition 65, which can be viewed here.

Although studies linking BHA to cancer primarily focus on animal data from the 1980s and 1990s, there are not many studies involving human subjects.

As part of its review, the FDA is issuing information requests, inviting both the public and industry to submit data regarding the use of BHA and its safety profile.

Health and Human Services Secretary Robert F. Kennedy Jr. stated, “This reassessment signifies the end of the ‘trust us’ era in food safety.”

This review is consistent with President Kennedy’s “Make America Healthy Again” policy, which aims to reduce harmful chemicals present in the food supply.

Last year, President Kennedy announced intentions to eliminate all artificial colors from the food supply by the year’s end, citing claims that these colors contribute to behavioral issues in children, including hyperactivity. The FDA notes this connection is monitored but not established.

In response, the FDA has approved more extensive use of “natural” dyes such as beetroot red and spirulina extract, a color additive sourced from algae.

Marion Nestle, a professor emeritus at New York University specializing in nutrition and public health, expressed her desire to understand how the FDA plans to assess the safety of BHA.

Nestle noted that previous toxicity studies on BHA largely depended on laboratory tests and animal studies, which may not effectively translate to human health outcomes.

She added that conducting research directly on human subjects would be impractical, costly, and ethically challenging.

Despite these challenges, Nestle commended the FDA’s decision to initiate a new safety review of BHA, highlighting that it has been on the public interest bureau’s “avoid” list for years, an organization that tracks food safety.

“It’s time for the FDA to address it,” said Nestle. “It will be intriguing to see what the reviewers conclude.”

As of now, the Consumer Brands Association, an industry group, has not responded to requests for comment.

Source: www.nbcnews.com

FDA Fast-tracks First Inhalable Gene Therapy for Cancer Treatment

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Inhaled Gene Therapy for Lung Cancer

Innovative Gene Therapy Delivered as a Mist for Lung Cancer Treatment

Nico De Pasquale Photography/Getty Images

An innovative inhaled gene therapy targeting lung cancer is rapidly advancing toward potential approval following encouraging results from clinical trials.

Dr. Wen Wee Ma at the Cleveland Clinic highlighted the findings at a recent American Society of Clinical Oncology conference in Chicago, stating, “Very encouragingly, this proves our hypothesis that the lung tumor actually shrunk.”

This groundbreaking treatment employs a virus to introduce immune-boosting genes into lung cells, enhancing their natural ability to combat tumors. Unlike traditional gene therapies, which often replace defective genes, this method focuses on modifying existing lung cells.

The unique inhalation delivery method represents a significant advancement in cancer treatment. “This is a completely different approach to anti-cancer treatment,” said Ma. Directly targeting the lungs enhances the efficiency and effectiveness of the treatment, particularly since lung cancer is notoriously difficult to treat with standard oral or intravenous therapies.

The therapy utilizes a harmless, modified herpes virus to inject two critical genes into lung cells: interleukin-2 and interleukin-12. These proteins, naturally produced by the body, help inhibit tumor growth. Unfortunately, tumors often diminish their effectiveness, necessitating the need for gene therapy to restore their production.


Since 2024, clinical trials have been ongoing with patients suffering from advanced lung cancer who have exhausted all other treatment options. The therapy is administered via a fine mist inhaled directly into the lungs.

At the oncology conference, Ma reported that the gene therapy has successfully reduced lung tumor sizes in three out of eleven trial participants, while also halting growth in another five. Although some patients reported side effects, such as chills and vomiting, no severe safety issues were noted.

Based on these promising outcomes, the U.S. Food and Drug Administration recently granted “Regenerative Medicine Advanced Therapy Designation” to the gene therapy, facilitating expedited approval processes for patient access.

However, it is important to note that this gene therapy is specifically designed for lung tumors and does not address tumors that have metastasized to other body parts. To expand its efficacy, Ma and his team are exploring combinations with immunotherapy and chemotherapy in a trial involving approximately 250 patients.

Crystal Biotech, the developer of this gene therapy, previously introduced the first FDA-approved gene therapy targeting the skin, using a similar modified herpes virus to treat patients with recessive dystrophic epidermolysis bullosa, a rare skin condition. The company is also developing inhaled gene therapies for cystic fibrosis and alpha-1 antitrypsin deficiency, both inherited lung diseases.

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Source: www.newscientist.com

FDA Warns Walmart Shrimp May Have Been Exposed to Radioactive Materials

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The Food and Drug Administration announced on Tuesday that consumers should refrain from purchasing certain frozen shrimp available at Walmart due to potential contamination with radioactive materials.

According to health officials in a recent news release, the Indonesian company involved is Pt. Indonesia’s Bahari McMur Sejati, commonly referred to as BMS Food.

A variety of raw frozen shrimp products processed by Indonesian firms can be found in Walmart locations across 13 states, including Alabama, Arkansas, Florida, Georgia, Kentucky, Missouri, Ohio, Pennsylvania, Texas, and West Virginia, as stated by the FDA.

The affected product includes Walmart’s “Great Value Brand Frozen Shrimp,” according to the health agency.

“If you have recently bought fresh frozen shrimp from Walmart that fits this description, please dispose of it,” the FDA advised. “Do not consume or serve this product.”

Health officials recommend that individuals speak with health care providers if they suspect they have been exposed to heightened levels of contaminants.

Both Pt. Bahari Makmur Sejati and Walmart did not respond promptly to requests for comments.

CS-137 is a radioactive isotope of cesium, a soft, pliable silver-white metal utilized in medical devices and gauges that liquefies at room temperature. As noted by the Environmental Protection Agency.

Repeated low-dose exposure to CS-137 “may raise the risk of cancer due to damage to DNA within living cells,” health officials stated in the news release.

The FDA mentioned that the US Customs and Border Patrol had alerted health agencies regarding the detection of CS-137 in shipping containers at ports in Los Angeles, Houston, Miami, and Savannah, Georgia. All containers that tested positive for CS-137 were denied entry into the country.

Health officials further noted that CS-137 was not found in products exceeding current Derived Intervention Levels for CS-137, set at 1200 BQ/kg.

However, the FDA stated, “The detected levels in the breaded shrimp samples could pose potential health risks.”

Matt Raviet

Matt Lavietes is a reporter for NBC News.

Austin Maren I contributed.

Source: www.nbcnews.com

FDA Scientists Make Progress at Food Safety Laboratory

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Officials from federal health agencies have decided to reverse the ruling that led to the dismissal of numerous scientists at the Food Safety Labs. They are also reviewing whether other critical positions have been affected.

A representative from the Department of Health and Human Services confirmed the reinstatement of these employees and mentioned that several individuals will also be returned to the office responsible for handling freedom of information requests.

In recent months, approximately 20% of FDA positions have been cut, marking one of the most significant workforce reductions among all agencies impacted by the Trump administration.

An HHS spokesperson stated that the departures were misleading due to erroneous employment codes.

In light of contradictory statements from FDA Commissioner Dr. Marty McCurry during a recent media interview, the decision to rehire scientists researching food-related illnesses and product safety—such as infant milk powder—will follow shortly.

“You could argue that no cuts were made to scientists and inspectors,” Dr. McCurry stated during Wednesday’s CNN broadcast.

Contrarily, many scientists were laid off from food and drug safety labs nationwide, including Puerto Rico, and from the veterinary unit working on avian flu safety. Employees on leave indicated that scientists in the tobacco sector, who were let go in February, including those examining the health implications of vaping, have not been considered for paid leave and reinstatement.

It remains uncertain how many dismissed employees will be permitted to return.

According to a department spokesperson, about 40 employees from Chicago’s Moffett Lab and a lab in the San Francisco area are being offered positions. Researchers in these facilities investigate various facets of food safety, including how chemicals and bacteria permeate food packaging and methods to ensure safety for infant formula. Some scientists in Chicago have also analyzed the findings of other labs to ensure the safety of milk and seafood.

Dr. Robert Caliph, the FDA commissioner under President Joseph R. Biden, described the term “decapitation and visceral withdrawal” as fitting for the abrupt loss of agency expertise. He noted that the FDA is already behind in meetings designed to assist businesses in developing safe products.

“Much of that involves routine daily tasks that significantly affect overall safety, though they’re not particularly controversial,” he commented. “It just requires effort, and they need personnel present to carry out their duties.”

Dr. McCurry also mentioned that the layoffs do not impact product reviewers or inspectors. However, their responsibilities are being hindered by voluntary departures, the reduction of support staff, and widespread disruptions at agencies, as many are looking to exit, according to former employees.

Hundreds of drug and medical device reviewers, representing about a quarter of the agency’s workforce, have opted out of major projects. As discussed on CNBC. Under FDA Ethics Rules, staff participating in employment interviews are prohibited from conducting agency reviews on products from firms seeking employment.

Dr. Gottlieb characterized the job cuts as “deep,” impacting the Bureau of Policy’s ability to process which drug brands can be offered as low-cost generics. Approvals for generic drugs could potentially save consumers billions.

The reduction in support staff overseeing inspections at food and drug facilities abroad has raised security concerns. Many of those who lost their positions were responsible for surveillance, ensuring inspectors’ safety, especially in hostile regions.

Source: www.nytimes.com

RFK Jr. criticizes FDA for banning alternative remedies and condemns drug industry’s influence

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In a speech aired on the Food and Drug Administration’s Maryland campus Friday morning, Robert F. Kennedy Jr. introduced himself as the country’s health secretary in a mean speech that touched on everything from the raptors of Lake Erie to the CIA.

Kennedy told agency staff in an effort to boldly avoid the impulse to protect the companies they regulate amid the pain of losing 20% ​​of the workforce under an overhaul of the health and human services sector.

Layoffs, voluntary departures and cuts in funding have already stopped the sectors controlling tobacco surveillance, drug approval processes, testing bird milk and bird flu cheeses, and food safety, which monitors and protects consumers from foodborne diseases.

In his remarks Friday, Kennedy suggested that the agency did not approve “alternative drugs” because of its subordination to wealthy businesses. Agent veterans argue that alternative products often fail to pass safety and efficacy standards.

He previously accused the FDA of suppressing raw milk, ivermectin and stem cell therapy.

He urged staff to resist the temptation to serve small groups of wealthy businesses at the expense of public health.

“We want to break away from it so that we can make our children healthy,” he said, according to a transcript of the speech shared with the New York Times. At another point, he said, “The deep nation is the real thing.” This is a light-journal reference to the vast federal bureaucracy that President Trump accused of as an obstacle to achieving his goals in his first term.

Department of Health and Human Services spokesman Andrew Nixon did not immediately respond to requests for comment on Kennedy’s remarks.

Kennedy also calls the FDA “sock dolls.” He used it in the past. Dynamics rewards “a very powerful incumbent in the industry,” he said at another time.

Drugmakers have benefited from a series of efforts by the FDA to speed up specific drug approvals and encourage businesses to develop drugs for serious illnesses that lack treatment. An FDA official said the program is intended to help patients.

The FDA has faced criticism over the past few years for several well-known drug approvals. For example, when granting approval for Alzheimer’s and Duchenne muscular dystrophy products, the top officials rejected the agency’s scientist or advisor.

Kennedy urged FDA employees to speak up if their boss greenlights products with insufficient evidence. “If your boss is making a mistake, if they approve something that shouldn’t be approved, we want to hear,” he said.

New FDA committee member Dr. Marty McCurry introduced Kennedy at a meeting Friday, supporting the goal of shaping healthier food supplies. He admitted that for some staff, cutting at the agency is “struggling with the ground.” He said the change was “to be integrated, more efficient and create more teamwork.”

Kennedy and Dr. McCurry were broadcast on video that aired on the agency White Oak campus outside Maryland.

Kennedy visited her father, Attorney General Robert F. Kennedy, at Washington’s Department of Justice, and recalls her child watching the Peregrine Falcons nest in the cupola of an old post office building. He also discussed his experiences at the Special Olympics, where he played the role of “Hugger” and coaching, playing the battles he played as an environmental lawyer.

Kennedy also complained about the rules governing the agency’s food department, which allow businesses to recognize that they can generally be recognized as being safe. This scale initially covered ingredients such as salt and vinegar to be acceptable in food without review. However, since then, thousands of ingredients have been added to the food supply without notice or testing by agents.

Food companies must provide a review of the materials to the FDA inspector on the premises, but such inspections can be performed once every five years. Kennedy is calling for an end to allow food companies to self-certify that the ingredients are safe.

“We literally don’t test chemicals before they’re added to food,” he said, according to the transcript. “Everything is engraved by the industry, as is generally perceived as safe.”

He went on to attribute the country’s diabetes rate to a loophole, adding that sugar also plays a role.

The speech was reminiscent of a social media message Kennedy posted in October, accusing the FDA of “a war with public health.” He said he is engaged in a “active suppression” of a series of unproven or unsafe products, including raw milk, chelate compounds, ivermectin, and “others that advance human health and cannot be patented by pharma.”

Here’s the post: “If you’re working for the FDA and are part of this corrupt system, you have two messages.

The agency is still shaking from thousands of job openings and voluntary deviations in the weeks since Kennedy was appointed health secretary. FDA employees who left in recent weeks include staff looking for drugs for byproducts that could cause cancer, and others working with international food safety staff to stop contaminated products from entering the United States.

The cuts in some regions are so deep that former FDA officials have suggested that the pharmaceutical industry could endanger billions of dollars to pay agents to ensure that the drug approval process is properly staffed.

Drugmakers are worried about what Kennedy’s leadership means for their benefit. They are worried that agency cuts will slow down drug reviews, including starting clinical trials, and will add a delay to final approval.

Public letter Dozens of biotech investors and executives have signed the signing, and industry leaders say they are “deeply concerned about the current state of the agency and its future.”

“Some of us have already encountered regulatory challenges that the FDA considers to be the result of the loss of experienced staff,” the letter states.

Source: www.nytimes.com

Layoffs at the FDA could lead to higher drug prices and jeopardize food safety

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Health Secretary Robert F. Kennedy Jr. announced widespread cuts at federal health agencies, including the Food and Drug Administration, which eliminates overlapping services and paper pushers.

However, interviews with more than a dozen current and former FDA staff featured another photo of the widespread impact of layoffs that ultimately cut the agency’s workforce by 20%. Among them are experts who have navigated the maze of law to determine whether expensive drugs can be sold as low-cost generics. Lab scientists who tested food and drugs for contaminants or fatal bacteria. Veterinary department experts investigating avian flu infections. Researchers who monitored advertisements that were aired for false claims about prescription drugs.

In many areas of the FDA, no employee will support overseas inspectors at risk of processing their pay, submitting retirement or layoff documents, or making the most of their agency’s credit card. Even libraries of institutions that relied on subscriptions to medical journals where researchers and experts were now cancelled have been closed.

FDA’s new commissioner, Dr. Marty McCurry, appeared on Wednesday in a much-anticipated appearance at Maryland headquarters. He gave a speech outlining a wide range of issues in the health care system, including an increase in chronic diseases. Employees were not given a formal opportunity to ask questions.

Approximately 3,500 FDA employees are expected to lose employment under the cuts. A spokesman for Health and Human Services did not answer the question.

When the Trump administration ran its first round with the FDA in February, it thwarted a team of scientists who did the nuanced job of ensuring the safety of surgical robots and devices injecting insulin into diabetic children. Some of the layoffs and cuts described as arbitrary volition by former FDA officials have quickly reversed.

Dr. David Kessler, a former agent committee member on the pandemic response under President Biden and White House adviser, said the latest round of layoffs has been deprived of decades of important experience and knowledge from the institution.

“I think it’s devastating, coincidence, thoughtful and confused,” he said. “I think they need to be revoked.”

It remains uncertain whether any of the lost jobs will be restored by the regime. In the interview, 15 current and former staff members spoke on condition of anonymity, some of whom spoke and explained the expected layoffs and expected impacts on food, drugs and medical supplies, fearing unemployment or retaliation.

Source: www.nytimes.com

Top FDA Vaccine Official Resigns, Cites Kennedy’s Accusations of ‘Misinformation and Lies’

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Dr. Peter Marks, a top Food and Drug Administration vaccine official, resigned under pressure on Friday, stating that Robert F. Kennedy Jr.’s aggressive attitude towards vaccines was irresponsible and posed a risk to public health.

“It became clear that truth and transparency are not valued by the secretary, but instead he desires blind confirmation of his misinformation and lies,” Dr. Marks wrote to Sarah Brenner, the agency’s representative. He reiterated his sentiments in an interview, stating, “This individual does not prioritize truth. He prioritizes followership.”

Dr. Marks resigned after being called to the Department of Health and Human Services on Friday afternoon, where he was given the ultimatum of resigning or being terminated, according to sources familiar with the situation.

Dr. Marks headed the Biologics Evaluation and Research Center responsible for approving and monitoring vaccine safety, as well as a variety of other therapies including cell and gene therapy. He was viewed as a steady and reliable presence by many during the pandemic, despite facing criticism for being overly accommodating to businesses seeking approval for treatments with complex evidence of effectiveness.

Ongoing scrutiny of the FDA’s vaccine program clearly placed Dr. Marks at odds with the new health secretary. Since Kennedy took office on February 13th, he has issued a series of directives on vaccine policy. He has alarmed those concerned about his potential to leverage his government authority to advance his long-standing campaign asserting vaccines are highly detrimental despite overwhelming evidence of their life-saving impact worldwide.

“Undermining trust in a well-established vaccine that has met the FDA’s rigorous standards of quality, safety, and efficacy for decades is irresponsible and poses a significant risk to public health and our nation’s well-being and security,” Dr. Marks wrote.

For instance, Kennedy promoted the use of vitamin A as a treatment during a major measles outbreak in Texas, downplaying the importance of vaccination. He has surrounded himself with analysts tied to the anti-vaccine movement and is pursuing studies examining long-debunked theories linking vaccines to autism.

On Thursday, Kennedy announced plans to establish a vaccine injury agency within the Centers for Disease Control and Prevention. He emphasized that this initiative was a top priority and would bring the “gold standard of science” to the federal government.

An HHS spokesperson stated on Friday night that Dr. Marks would no longer have a place at the FDA if he did not commit to transparency.

In his resignation letter, Dr. Marks highlighted the tragic toll of measles amid Kennedy’s lukewarm approach to the urgent vaccination needs among many unvaccinated individuals in Texas and other states.

Dr. Marks pointed out that through widespread vaccine availability, “over 100,000 children who received vaccinations last year in Africa and Asia were saved.”

Dr. Marks expressed his willingness to address Kennedy’s vaccine safety and transparency concerns in public forums and through collaboration with national experts in science, engineering, and medicine, which he was rebuffed.

“I have exhausted all efforts to work with them to restore confidence in vaccines,” Dr. Marks stated in an interview. “It became evident that this was not their goal.”

With that, Dr. Marks bid farewell to the FDA.

“His leadership has been instrumental in driving medical innovation and ensuring life-saving treatments reach those in need,” stated Ellen V. Sigal, founder of the cancer research advocacy group Friends and a close associate of Dr. Marks. His departure, she noted, “will leave significant gaps.”

Dr. Marks guided the agency and its external advisors on the type of evidence required to pilot the FDA’s vaccine program amid the tumultuous year of the coronavirus pandemic and expedite emergency authorizations for vaccines developed under the Trump administration’s Operation Warp Speed.

In June 2022, he urged an external expert panel to consider the risks the virus posed to children under five years old, leading the panel to recommend the vaccine for that age group later that day.

“We must be cautious not to be paralyzed by the number of pediatric deaths due to the overwhelming number of fatalities we are facing here,” Dr. Marks cautioned at the time.

Dr. Peter Hotez, a vaccine expert at Baylor College of Medicine, spoke highly of his regular interactions with Dr. Marks during the pandemic, describing him as deeply committed to leveraging science to aid the American populace. “He was a pandemic hero, and it’s truly unfortunate to see him go,” Hotez remarked.

Dr. Marks faced skepticism from some within the FDA, including former members of his own vaccine team. Two senior regulators in the agency’s vaccine office resigned in 2021 over the Biden administration’s efforts to push for the approval of Pfizer’s COVID-19 vaccine and booster shot.

Kennedy’s call for further investigation into vaccine injuries was met with reservations by Dr. Michael Osterholm, director of the University of Minnesota’s Center for Infectious Disease Research and Policy, who noted that such research had been a focal point for decades. “I fear this is an attempt to magnify vaccine harm out of proportion to the actual risk,” Osterholm cautioned.

Dr. Marks shared these concerns, expressing his desire in his letter to mitigate the harm inflicted by the current administration.

“My hope,” he penned, “is that the unprecedented assault on scientific truths that has detrimentally impacted our nation’s public health will cease in the coming years, allowing our citizens to fully benefit from the wide array of medical advancements.”

Source: www.nytimes.com

FDA Urges Pet Food Companies to Review Safety Plans in Light of Bird Flu Outbreak

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The number of cats increasing that have died or become ill after consuming raw pet food and raw milk contaminated with the H5n1 virus has prompted health authorities to take special precautionary measures to protect pet food companies from bird flu. They are advising pet food makers to follow food safety plans such as sourcing ingredients from healthy flocks and applying heat treatments to inactivate viruses, as suggested in recent guidance from the Food and Drug Administration.

Since the H5n1 virus started spreading in 2022, there have been bird outbreaks under all conditions. Cats appear to be particularly susceptible to the H5N1 virus, with many household cats and wild cats becoming infected since its emergence in 2022. Some farm cats have fallen ill after consuming raw milk, while others have died after consuming contaminated raw pet food.

Despite the FDA guidance, some experts like Dr. Jane Cycks from the University of California, Davis School of Veterinary Medicine have raised concerns about the lack of detailed instructions on guaranteeing the absence of H5N1 in food. The FDA has advised pet owners to cook raw pet food to eliminate risks and follow USDA guidelines for safe food handling.

In response to the situation, some raw pet food companies have implemented safety measures such as sourcing quality ingredients and using processes like high-pressure pasteurization. However, experts emphasize that cooking is the only certain way to eliminate the risk of H5N1 in pet food.

Overall, both the Centers for Disease Control and Prevention and the American Veterinary Medical Association recommend against feeding companion animals raw or undercooked meat due to the potential risks associated with pathogens like H5N1.

While high-pressure pasteurization is advertised as a method to kill pathogens, experts caution that cooking to internal temperature is the most reliable way to ensure food safety. Consumers are advised to cook raw pet food thoroughly before feeding it to their pets to reduce the risk of transmission of bird flu.

For those who prefer raw pet food brands, experts suggest cooking the food before feeding it to ensure the safety of pets.

Source: www.nbcnews.com

FDA advises updating COVID-19 vaccine to target KP.2 variant

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The Food and Drug Administration on Thursday recommended that pharmaceutical companies develop coronavirus vaccines targeting the KP.2 strain, a descendant of the highly contagious JN.1 variant that began spreading widely in the United States this winter. The announcement comes just one week after an FDA advisory committee unanimously recommended updating COVID-19 vaccines in the fall to target the JN.1 variant or its descendants.

After the vote, there was disagreement between committee members and Dr. Peter Marks, the agency’s top vaccine regulator, over which strain the agency should choose. Most committee members expressed a preference for JN.1, but Dr. Marks favored choosing a newer strain like KP.2. “We’re paying an incredibly high premium for the mRNA vaccines to get the freshest vaccine,” Marks said, likening getting the vaccine to buying milk at the store.

The FDA said it had initially advised drugmakers on June 6 to target the JN.1 variant, but the agency continues to monitor circulating strains and “based on the latest available data and recent increases in COVID-19 cases in areas of the country, the FDA has further determined that the JN.1 lineage is preferred for improved vaccines,” with the KP.2 strain preferred “when possible,” the FDA said.

The JN.1 is hardly in circulation in the United States. Centers for Disease Control and Prevention As of Saturday, KP.2 accounted for 22.5% of new coronavirus cases in the U.S., while its sister variant, KP.3, accounted for 25% of new cases.

The FDA’s decision allows pharmaceutical companies to begin manufacturing and distributing the vaccine, which is expected to be used as part of COVID-19 vaccination campaigns this fall.

Three pharmaceutical companies, Pfizer, Moderna, and Novavax, are producing coronavirus vaccines. Pfizer and Moderna’s vaccines are mRNA-based, while Novavax’s vaccine is protein-based. Because protein-based vaccines take a long time to produce, Novavax has indicated that it will not be able to produce the KP.2 vaccine by the fall. Instead, it plans to distribute the JN.1 vaccine that it has already produced.

This is the third time the vaccine has been updated to target circulating strains. The process of selecting the next vaccine is becoming more routine, similar to the annual influenza vaccination update, with vaccinologists selecting the strains in the spring and then conducting vaccination campaigns in the fall.

At the advisory committee meeting, the pharmaceutical company presented data showing that compared to current vaccines that target the variant XBB.1.5, which is not currently in circulation, the JN.1 vaccine should generate higher levels of antibodies against circulating virus strains.

The committee did not recommend who should get the latest vaccine, leaving that to the CDC, which will be holding its own advisory committee meeting later this month.

Source: www.nbcnews.com

The FDA Approves Pfizer’s First Gene Therapy for a Rare Inherited Bleeding Disorder

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The Food and Drug Administration approved Pfizer’s treatment of rare genetic diseases and bleeding disorders, making it the company’s first-ever gene therapy. The approval came on Friday. This groundbreaking gene therapy, named Bekvez, is for adults with moderate to severe hemophilia B, meeting specific requirements. Pfizer spokesperson mentioned that the medication will be available by prescription to eligible patients this quarter with a hefty price tag of $3.5 million, making it one of the most expensive drugs in the United States.

More than 7,000 people in the US live with hemophilia B, primarily affecting men as it causes a lack of Factor IX, a crucial protein for blood clotting. Bekvez is designed as a one-time treatment to enable patients to produce their own Factor IX to prevent and control bleeding effectively.

Pfizer’s approval for Bekvez is a major milestone in the company’s efforts to diversify its portfolio after facing challenges due to the decline in its COVID-19 business. The company has heavily invested in anti-cancer drugs and gene and cell therapies, aiming to revolutionize the treatment of chronic diseases. Pfizer also acquired the rights to manufacture and market Bekvez in collaboration with Spark Therapeutics.

In addition, Pfizer is seeking FDA approval for marstasimab, an experimental antibody to treat hemophilia A and B. The company is also developing a gene therapy for Duchenne muscular dystrophy. Bekvez will be competing with similar gene therapies like Hemogenix from CSL Behring, which also comes with a significant price tag of $3.5 million in the US, marking a new era in the treatment of hemophilia.

Despite the high costs and logistical challenges associated with gene therapies, medical experts are hopeful that these one-time treatments can replace traditional lifelong treatments for chronic diseases, offering long-term benefits to patients.

Source: www.nbcnews.com