Latest news with #VinayPrasad
Yahoo
5 days ago
- Business
- Yahoo
Intellia stock dips following safety event in Phase III gene therapy trial
Intellia Therapeutics' stock has fallen nearly 23% following an announcement that a patient in its Phase III trial for a gene therapy targeting transthyretin amyloid (ATTR) cardiomyopathy suffered a serious adverse event (AE). In a filing by the Securities and Exchange Commission (SEC), the company reported that one patient experienced Grade 4 liver transaminase elevations after treatment with nexiguran ziclumeran (nex-z) in the Phase III MAGNITUDE trial (NCT06128629). According to Intellia, the event is resolving without the need for hospitalisation or medical intervention. Upon the announcement, Intellia's stock, listed on the Nasdaq exchange, dropped by 22.87% from $9.66 a share at market close on 28 May to $7.45 at market close on 29 May. Intellia emphasised that this is a single event from more than 200 patients dosed with nex-z in the MAGNITUDE study. Other AEs have been similar to those seen in the Phase I study, including infusion-related reactions and asymptomatic liver transaminase elevations. The SEC filing added that Intellia said it will continue to monitor these events as the study progresses. The trial has so far enrolled 365 patients out of an expected total enrollment of approximately 765 patients, which Intellia says is on track. The company aims to complete enrollment by early 2027. Intellia is also investigating nex-z in the Phase III MAGNITUDE-2 study (NCT06672237), which is enrolling patients with hereditary ATTR amyloidosis with polyneuropathy (ATTRv-PN). Dosing has started, with investigators seeking data on whether the therapy will lead to a reduction in serum TTR. Intellia will be waiting for data from MAGNITUDE-2 before filing a biologics licence application (BLA) to the US Food and Drug Administration (FDA) in 2028, with hopes for a 2029 commercial launch. It has been a challenging time for gene therapy development, with several severe AEs and, in some instances, fatalities seeming to plague investors' views on the therapeutic approach. On 27 May, Rocket Pharmaceuticals announced that a patient died in its Phase II trial for a Danon disease gene therapy after suffering a serious AE. In March 2025, Sarepta and Roche reported the death of a teenage patient dosed with its AAV gene therapy for Duchenne muscular dystrophy (DMD), Elevidys (delandistrogene moxeparvovec-rokl), causing agencies to pause ongoing clinical studies. This pause has since been lifted. Last year, a fatality in Pfizer's Phase II DMD gene therapy trial, as well as low efficacy in a Phase III study, caused the big pharma to drop the development of the AAV candidate, named fordadistrogene movaparvovec. Earlier this month, the FDA shared that American haematologist oncologist Vinay Prasad will lead its Center for Biologics Evaluation and Research (CBER), the division responsible for regulating gene therapies and vaccines. Prasad has openly criticised Elevidys, primarily about the clinical evidence and the FDA's decision to approve the therapy, which raises questions about his overall opinion of the gene therapy approach. "Intellia stock dips following safety event in Phase III gene therapy trial" was originally created and published by Clinical Trials Arena, a GlobalData owned brand. The information on this site has been included in good faith for general informational purposes only. It is not intended to amount to advice on which you should rely, and we give no representation, warranty or guarantee, whether express or implied as to its accuracy or completeness. You must obtain professional or specialist advice before taking, or refraining from, any action on the basis of the content on our site. Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Yahoo
6 days ago
- Business
- Yahoo
FDA's Surprising COVID Vaccine Change Impacts Millions—What You Need to Know
The FDA has updated COVID-19 vaccine regulations. Only people aged 65 and up, along with certain groups, will be eligible to receive booster shots. FDA officials are asking for more data around the vaccine's effectiveness for people who are otherwise healthy. The U.S. Food and Drug Administration made a surprising move last week, announcing that the agency will limit the use of the COVID-19 vaccine to certain groups. The changes are expected to go into effect in the fall. Just this week, the Centers for Disease Control and Prevention (CDC) will no longer recommend that pregnant people and healthy children get the COVID-19 vaccine (note: The CDC still lists pregnant people as high-risk for COVID-19 complications—more on this below). The new regulations were laid out in an article published in The New England Journal of Medicine titled, 'An Evidence-Based Approach to Covid-19 Vaccination.' In the article, FDA vaccine division chief Vinay Prasad, M.D., and FDA commissioner Martin Makary, M.D., wrote that the American policy with COVID-19 booster shots is the 'most aggressive' compared with policies in European nations. 'The U.S. policy has sometimes been justified by arguing that the American people are not sophisticated enough to understand age- and risk-based recommendations,' the article reads. 'We reject this view.' The FDA usually authorizes or approves vaccines for use, while the Centers for Disease Control and Prevention (CDC) specifies recommendations on how they should be used under the guidance of the Advisory Committee on Immunization Practices (ACIP). The FDA dictating how vaccines should be used is a different approach, points out William Schaffner, M.D., an infectious disease specialist and professor at the Vanderbilt University School of Medicine. Meet the experts: Thomas Russo, M.D., professor and chief of infectious disease at the University at Buffalo in New York, William Schaffner, M.D., an infectious disease specialist and professor at the Vanderbilt University School of Medicine; infectious disease specialist Amesh Adalja, M.D., senior scholar at Johns Hopkins University Center for Health Security The change seems to be political, given that there is a new administration in place and no new data has been released on side effects, safety, or effectiveness of these vaccines. But what are the new FDA COVID vaccine regulations, and what does this mean for the future of COVID care? Here's the deal. Until now, government health agencies have recommended that everyone over the age of 6 months get an annual COVID-19 vaccine. The vaccine is tweaked each year to try to match up with circulating strains of the virus and to provide the most protection. But the new regulations will limit the use of the vaccine to people aged 65 and up, along with people over the age of 6 months who have certain medical conditions. The list of qualifying medical conditions is long, and includes more obvious things like asthma, cancer, pregnancy, and chronic lung diseases. But it also includes things like physical inactivity and mood disorders, opening the vaccine up to a wide range of people. The FDA may now require more studies before approving COVID-19 vaccines for healthy Americans under the age of 65, according to the paper. The article makes the argument that these changes are more in line with what the rest of the world is doing. 'All other high-income nations confine vaccine recommendations to older adults (typically those older than 65 years of age), or those at high risk for severe COVID-19,' the FDA officials write. 'The United States has adopted a one-size-fits-all regulatory framework and has granted broad marketing authorization to all Americans over the age of 6 months.' 'The strategy in the paper aligns with what other countries have done with updated COVID vaccines in the current context of a heavily immune population,' says infectious disease specialist Amesh Adalja, M.D., senior scholar at Johns Hopkins University Center for Health Security. 'It importantly demarcates high-risk from low-risk individuals when it comes to how to think about the value of COVID vaccines and what types of studies are most likely to answer questions about that value in these populations.' Other than those over 65 years of age, the FDA specifically flagged these groups and conditions, among others not listed, as qualifying for the COVID-19 vaccine in the future: Asthma Cancer Cerebrovascular disease Chronic kidney disease Chronic lung disease Chronic liver disease Cystic fibrosis Diabetes (type 1 and type 2) Gestational diabetes Disabilities, including Down syndrome Heart conditions HIV Mental health conditions Dementia Parkinson's disease Obesity Physical inactivity Pregnancy and recent pregnancy Primary immunodeficiency Smoking, current and former Solid organ or blood stem-cell transplantation Tuberculosis Use of corticosteroids or other immunosuppressive medications Note: While the CDC still specifies pregnancy as a condition that puts people at high risk for COVID complications, the CDC will no longer recommend routine COVID shots for pregnant women and healthy children, Health and Human Services Secretary Robert F. Kennedy Jr. recently announced on X. Yes, the COVID vaccines are still considered safe. This paper did not conduct or call out any new research around these vaccines. 'This vaccine is extremely safe,' says Thomas Russo, M.D., professor and chief of infectious disease at the University at Buffalo in New York. According to the CDC, the most common side effects of both Pfizer and Moderna vaccines include pain at the injection site, fatigue, headache, muscle and joint pain, and fever. 'During the COVID-19 pandemic, COVID-19 vaccines underwent the most intensive safety analysis in U.S. history,' the CDC's website reads. 'COVID-19 vaccines continue to be monitored for safety, even after FDA approval, to make sure they continue to meet FDA's standards for safety and effectiveness.' There hasn't been recent data on the effectiveness of the COVID-19 vaccines, but data from clinical trials when the vaccines were first released (which now dates back five years) showed that the Pfizer vaccine was 95% effective at preventing COVID-19, while the Moderna vaccine was 94.1% effective. Studies conducted on the Novavax vaccine found that it was 90% effective at preventing people from developing the virus and 100% effective at protecting people from severe disease. There is a mixed reaction, although doctors as a whole don't seem shocked. 'We have to look at the reality: In 2024-2025, only a very small portion of the population availed themselves of the vaccine,' Dr. Schaffner says. As a result, he says that changing the requirements to get the vaccine is unlikely to have a big impact on the population. 'If the goal is to prevent severe disease, targeting vaccines to high-risk individuals is the optimal strategy,' Dr. Adalja says. 'For lower risk individuals, the goal has always been less clear as protection against infection is transient and as they don't have a risk for severe disease, the cost-effectiveness of a vaccine that, at this stage, chiefly protects against severe disease doesn't apply.' But Dr. Adalja says that the initial COVID-19 vaccine series 'should be part of routine childhood immunization.' He also points out that it's 'unclear' if new clinical trials will be needed to show that there's value for young children to receive the vaccine. Dr. Russo has concerns that the new regulations seem to have a 'double standard that's not based in science' for the COVID-19 and influenza vaccines, both of which target viruses that change seasonally. 'Interestingly, this does nothing for the influenza vaccination, and we get annual boosters for that to target the circulating strains,' he says. 'They seem to think it's fine for influenza but, when you compare it to influenza, COVID remains a more severe disease.' It's not clear at this point if the FDA plans to alter regulations around other vaccines in the future. If you want to get the COVID-19 vaccine in the future but you don't fall into a vulnerable group, doctors say you probably still can. 'It will likely be the case, as it is for other vaccines, that they can be administered off-label to those who want them—with an out-of-pocket charge,' Dr. Adalja says. Dr. Russo points out that the criteria for getting the vaccine is 'fairly liberal,' though. 'The way this is phrased, most Americans can make claims to this vaccine if they really want it,' he says. Again, this is an unusual approach to recommendations around vaccination in the U.S. But the next meeting of the ACIP is in late June, and will likely address next steps on how the COVID vaccine will be used. 'We just have to wait until June,' Dr. Schaffner says. As of this second, though, the CDC still recommends that everyone over the age of 6 months get vaccinated against COVID-19. This article is accurate as of press time. However, as guidelines evolve, some of the information may have changed since it was last updated. While we aim to keep all of our stories up to date, please visit online resources provided by the CDC, WHO, and your local public health department to stay informed. Always talk to your doctor for professional medical advice. You Might Also Like Can Apple Cider Vinegar Lead to Weight Loss? Bobbi Brown Shares Her Top Face-Transforming Makeup Tips for Women Over 50
Medscape
27-05-2025
- Health
- Medscape
FDA Advisers Recommend No Changes to COVID Vaccine for Fall
US Food and Drug Administration (FDA) advisers recommended that manufacturers make no changes to the COVID-19 vaccine that will be available for boosters later this summer. The Vaccines and Related Biological Products Advisory Committee (VRBAC) voted 9-0 that the 2025-26 vaccines should stay the same, containing a single JN.1 strain, which has been dominant globally since 2024. 'There is efficacy,' said Panel Member Eric J. Rubin, MD, PhD, editor-in-chief of The New England Journal of Medicine and adjunct professor of immunology and infectious diseases at the Harvard T.H. Chan School of Public Health, Boston. 'It's not fantastic. It's not what it was originally,' he said, adding but that in a population that has broad immunity to the SARS-COV-2 virus, 'it wasn't bad.' The agency usually follows its panels' advice, but nothing is certain for the vaccines this fall. As reported by Medscape Medical News , FDA Commissioner Martin Makary, MD, and FDA Center for Biologics Evaluation and Research Director Vinay Prasad, MD, in a 'town hall' on May 20 and in a paper in TheNew England Journal of Medicine said the agency planned to start requiring randomized, controlled trials for approval of COVID vaccines in healthy Americans who are younger than 65 years. Approvals of boosters over the last 4 years have been based on observational data, and the vaccines have been recommended for all Americans aged 6 months or older. The FDA has not held any further meetings nor issued a final rule or policy to clarify whether this year's COVID vaccine boosters would be required to conduct new trials. The agency may have signaled where it is headed with its May 17 full approval for Novavax's COVID-19 vaccine, which previously only had emergency use authorization. The FDA limited the vaccine's use to adults older than 65 years and anyone older than 12 years at high risk. Some panelists were concerned that a randomized trial requirement might cause delays. 'Is there a possibility that if we choose a different vaccine [strain], it is actually going to be different regulatory measures that are taken that would delay the vaccine getting to the market?' asked Stanley M. Perlman, MD, PhD, chair of microbiology and immunology at Carver College of Medicine, University of Iowa, Iowa City, Iowa. Rubin said he believes the observational data presented by COVID vaccine makers 'has a lot more richness,' than a randomized trial, given the various exposures to illness and vaccines in a widely varied population. 'I don't think the randomized, controlled trial is feasible,' said Rubin. David C. Kaslow, MD, director of the Office of Vaccines Research and Review at FDA, said that although the agency is still discussing how it will regulate COVID vaccines, 'I think there's a shared desired outcome for the timely availability of vaccines to prevent COVID-19.' Kaslow said the proposed new regulatory framework could be a 'potential topic for a future VRBPAC meeting.' Representatives from Pfizer, Moderna, and Novavax all said they would have vaccines ready for the fall. 'We look forward to working with the agency to ensure timely provision of appropriate data to support COVID-19 vaccine approval for those who need it most,' said Kayvon Modjarrad, MD, PhD, executive director of Viral Vaccines and Immunology Vaccine Research and Development at Pfizer. Continuing Burden, Low Vaccine Uptake At the meeting, US Centers for Disease Control and Prevention (CDC) officials and vaccine makers said that COVID-19 is still dangerous, especially for those at highest risk, primarily individuals older than 75 years and children younger than 6 months. The CDC estimates that 30,000-50,000 people have died from COVID since October. Few Americans choose to get a COVID vaccine, hovering around 25% for adults aged 18 years to 64 years, rising to 50% for those older than 75 years, said Ruth Link-Gelles, PhD, MPH, of the CDC's National Center for Immunization and Respiratory Diseases. Vaccine effectiveness against hospitalization and critical illness in immunocompetent adults older than 65 years was around 40%-50%, said Link-Gelles. It was the same or slightly lower for immunocompromised adults in the same age group. Pfizer estimated that COVID vaccines prevented some 107,000 hospitalizations and 6700 in-hospital deaths in 2024. The vaccine makers argued that their products are safe and effective. 'Our vaccines are among the most extensively-monitored products licensed,' said Pfizer's Modjarrad. The FDA, however, has recently instructed Pfizer and Moderna also to update labeling to expand a warning about myocarditis and pericarditis. Pfizer's label warned of an elevated risk in men 12- to 17-year-olds, Moderna's in men 18- to 24-year-olds. Both will now cite an elevated risk for men aged 16 years to 25 years. In its letter, the agency cited data from a Lancet study of patients with COVID-19- vaccine–associated myocarditis 'showing persistence of abnormal cardiac magnetic resonance imaging findings that are a marker for myocardial injury at a median follow-up of approximately 5 months.' Some Call for Withdrawal, Others for More Access During the hour-long public hearing portion of the meeting, most speakers asked the FDA to immediately recall all COVID vaccines, citing a variety of harms — many of them unproven. 'Rewind now and pull these shots,' said Brian Hooker, PhD, the chief scientific officer of Children's Health Defense — the organization formerly run by current Health and Human Services Department Secretary Robert F. Kennedy, Jr. Hooker cited myocarditis as a concern. 'Based on this alone, the product should have been completely withdrawn,' said Hooker. He also said that COVID vaccines 'have been also strongly associated with clotting disorders, including stroke, pulmonary embolism,' and other disorders. Kaitlin Sundling, MD, PhD, assistant professor of pathology and laboratory medicine at the Wisconsin State Lab of Hygiene, said COVID vaccines should be more widely available. 'All people, regardless of age and medical history, need broad access to regularly updated COVID vaccine formulations,' said Sundling, who added she was concerned about the FDA's potential requirement for randomized trials and to limit vaccine approvals to those older than 65 years or at high risk. Noting that the panel had essentially been asked to not talk about that proposed new COVID regulatory framework at the meeting, Sundling said 'There is a very real concern that this committee is being used as a pawn to eliminate vaccine access for the US population.'
Japan Times
26-05-2025
- Health
- Japan Times
The FDA's new COVID-19 vaccine policy is clear as mud
When news broke that the U.S. Food and Drug Administration was rolling out a new plan for COVID-19 vaccine approvals, my phone instantly started buzzing. Friends, family members and colleagues all had the same question: Does this mean I can't get a fall booster? I wish I had a straightforward answer for them. Despite an explanation of the new strategy published last week from FDA Commissioner Marty Makary and Vinay Prasad, who oversees vaccines at the agency, public health experts were left guessing about basic details such as how it will affect access to COVID-19 shots. Do healthy people have a choice or not? What is the status of shots for kids? For an agency that has vowed to build the public's trust by offering transparency and an open dialogue, it was a frustrating start. Here's what we know: The FDA is adopting a risk-based strategy for approving new COVID-19 vaccines that will limit their use to people 65 and older or anyone with a health condition that puts them at risk for severe disease — a group Prasad and Makary estimate accounts for about 100 million Americans. The new policy keeps the current standards for greenlighting vaccines, meaning pharmaceutical companies need only to produce data showing theirs prompts people to produce antibodies against the virus. For healthy adults and children, new vaccines, including boosters updated to match circulating strains of the virus, seem to be on ice. FDA officials will require drug manufacturers to run studies proving their vaccines are better than a placebo in preventing symptomatic infections in people ages 50 to 64 — a group Prasad said was chosen because health agencies around the world express the most disagreement over the benefits of vaccination for the demographic. They said companies can choose to pursue studies that demonstrate their drugs effects in other groups. The future of pediatric COVID-19 vaccines seems particularly foggy, since updated shots also function as the primary series for unvaccinated children. While billed as "a balance of regulatory flexibility and a commitment to gold-standard science,' the policy seems designed to put the vaccines out of reach. Let's start with the feasibility and ethics of running the trials. Who would volunteer for such a study? COVID-19 skeptics or anyone hesitant about the safety of the vaccine seem unlikely to participate. Why would anyone who trusts the evidence that the vaccine is safe and effective — and that the risks from COVID-19 are real — want to take a chance on getting a placebo, says Paul Offit, director of the Vaccine Education Center at Children's Hospital of Philadelphia. "This is a low-risk group, but it's not a no-risk group,' he says. Although we are no longer in an emergency, COVID-19 hasn't gone away. Nearly 1,400 Americans died of the disease last month — and we're in a lull. During last year's late-summer wave, more than 1,000 people were dying each week, according to data from the Centers for Disease Control and Prevention. The required trials would also be expensive. Thousands of people would need to be enrolled and, according to the FDA's plan, followed for six months. With few people opting for COVID-19 boosters these days, companies might decide the investment isn't worth it. If FDA's advisers, who meet this week to choose a formula for fall shots, ask drug companies to change the vaccine, millions of Americans could be left without a booster this fall. Vaccines are a public good. I am at low risk of a serious COVID-19 infection, but my motivation to get a booster is about more than my own health. When I roll up my sleeve, I am thinking about my elderly parents, a dear family member undergoing chemotherapy and all the other people in my life who are more vulnerable to this virus than I am. I know a booster can't guarantee that I won't get sick and pass that infection onto others, but the evidence that it lowers the chances of both, at least for a few months, is sufficient for me to believe it's worth getting. I should have that option. Prasad dismisses that argument, noting in a town hall (an event where only Makary was allowed to ask questions) that there's "no high-quality evidence' to suggest getting a booster to "go visit grandma' is helpful. He and Makary argue that without data to support that claim, people are operating under a false sense of security. However, the CDC and others have offered evidence showing that higher vaccination rates in a community result in fewer infections and fewer people ending up hospitalized. It's true that finding comes from mining real-world data rather than randomized, placebo-controlled trials, but that doesn't mean people can't factor it into their choice to vaccinate. And Americans have spent the last several years making personal decisions with the knowledge that some information is murky, says Georgetown University professor Jesse Goodman, who previously served as the FDA's chief scientist. "I would argue that people, just as they are now, can make up their mind about whether with the imperfect data they'd like to get the vaccine,' he says. "I'd rather give people the choice.' Anyone worried about losing that choice should be just as concerned about how all of this is unfolding. The FDA's realm is evaluating the evidence about vaccines, while the CDC is charged with making recommendations about how they should be used. In nearly all cases, those decisions are based on advice from the CDC's Advisory Committee on Immunization Practices (ACIP), independent experts who carefully and publicly debate the evidence. "I'm very concerned that this really is the first step towards neutering ACIP,' says Michael Osterholm, director of the Center for Infectious Disease Research and Policy in Minnesota. "ACIP should be involved with this. This is not FDA's role to be making recommendations on vaccine for risk groups.' That committee will meet in June to discuss COVID-19 boosters. Ironically, they are likely to arrive at guidelines that largely align with Makary and Prasad's focus on the elderly and high-risk groups. Yet public health experts note the panel's recommendations are likely to differ in one notable way: Given the safety of the vaccine, they're likely to leave the door open for everyone to decide for themselves. Makary and Prasad argue that generating better data will drive more people to follow them — and that it's the lack of confidence that's holding individuals back. Public health experts have a different view. Many people simply don't see COVID-19 as the threat it once was and so are opting out. Others have been deterred by the intense rhetoric and misinformation about the shots — much of it coming from current health agency leadership. In an ideal world, we'd have perfect data that allows people to make easy decisions about their health. And while continuing to study our vaccines and adapt our recommendations to fit the evolving state of the outbreak is perfectly reasonable, it should be done through the normal channels — and communicated in terms that everyone can understand. Lisa Jarvis is a Bloomberg Opinion columnist covering biotech, health care and the pharmaceutical industry. Previously, she was executive editor of Chemical & Engineering News.
Yahoo
24-05-2025
- Business
- Yahoo
Moderna, Inc. (MRNA) Submits Updated COVID Vaccine for Regulatory Review
Moderna, Inc. (NASDAQ:MRNA) announced on Friday that it has submitted a marketing application to the U.S. Food and Drug Administration (FDA) for review of its updated COVID-19 vaccine. The revised version of the vaccine, marketed as Spikevax, follows FDA guidance recommending that this year's shots target variants from the JN.1 lineage, specifically favoring the LP.8.1 strain. Current government data shows LP.8.1 is responsible for about 70% of COVID-19 cases in the US. Moderna, Inc. (NASDAQ:MRNA), a pioneer in mRNA-based medicine, has been advancing treatments by combining science, health, and technology. The company gained prominence for developing one of the first and most effective COVID-19 vaccines during the pandemic. Moderna, Inc. (NASDAQ:MRNA) expects to roll out the new version of Spikevax by mid-August. The updated filing comes amid heightened regulatory oversight under new FDA leadership. Vaccine manufacturers now face stricter requirements, which could drive up development costs. Earlier this week, the FDA announced that companies will need to conduct new clinical trials for annual boosters aimed at healthy adults under 65, reserving approvals mainly for older or high-risk groups. Top US vaccine official Vinay Prasad said developers will be required to run placebo-controlled trials in adults aged 50 to 64, and will be encouraged to do the same in very young children. Moderna, Inc. (NASDAQ:MRNA) has not revealed the age groups its new vaccine is intended for or whether it conducted additional trials. Earlier this week, the company pulled its application for approval of its combined flu and COVID-19 vaccine candidate following discussions with the FDA. While we acknowledge the potential of MRNA to grow, our conviction lies in the belief that some AI stocks hold greater promise for delivering higher returns and have limited downside risk. If you are looking for an AI stock that is more promising than MRNA and that has 100x upside potential, check out our report about this cheapest AI stock. READ MORE: and Disclosure. None.
