Sarepta will resume gene therapy shipments after FDA review of recent patient death
The Food and Drug Administration said it recommended lifting the hold for young patients with Duchenne's muscular dystrophy who are still able to walk. Regulators had requested the pause after the deaths of two older teenagers who were taking the therapy. The FDA also said in a statement it determined that a recently reported death of an 8-year-old boy was unrelated to the therapy.
Company shares surged more than 16% after the announcement to $13.86 in afterhours trading. The jump is the latest in a series of drastic stock movements triggered by changing fortunes for the company's best-selling product.
Elevidys is the first gene therapy approved in the U.S. for Duchenne's muscular dystrophy, the fatal muscle-wasting disease that affects boys and young men, resulting in early death. It received accelerated approval in 2023 for a narrow range of young patients and was expanded last year for use in older patients, including those who can no longer walk.
The FDA decision Monday 'significantly improves Elevidys' sales outlook in the near-term,' Jefferies analyst Andrew Tsai told investors, in a note after the announcement. 'The street will feel relieved about the situation, suggesting meaningful stock upside potential.'
Sarepta's therapy has been under scrutiny from regulators after two teenage boys died earlier this year from acute liver injury, a known side effect of the treatment. The FDA then requested a pause in shipments of the drug after the death of a third patient taking a different Sarepta therapy.
FDA officials have suggested the company will need to provide new study data on safety to resume Elevidys' use in older patients.
'The FDA will continue to work with the sponsor regarding non-ambulatory patients, which remains subject to a voluntary hold, following two deaths,' FDA said in its statement.
___
The Associated Press Health and Science Department receives support from the Howard Hughes Medical Institute's Department of Science Education and the Robert Wood Johnson Foundation. The AP is solely responsible for all content.
Try Our AI Features
Explore what Daily8 AI can do for you:
Comments
No comments yet...
Related Articles
Yahoo
2 minutes ago
- Yahoo
NATO to coordinate regular and large-scale arm deliveries to Ukraine. Most will be bought in the US
BRUSSELS (AP) — NATO has started coordinating regular deliveries of large weapons packages to Ukraine after the Netherlands said it would provide air defense equipment, ammunition and other military aid worth 500 million euros ($578 million). Sweden also announced Tuesday it would contribute $275 million to a joint effort along with its Nordic neighbors Denmark and Norway to provide $500 million worth of air defenses, anti-tank weapons, ammunition and spare parts. Two deliveries of equipment, most of it bought in the United States, are expected this month, although the Nordic package is expected to arrive in September. The equipment is supplied based on Ukraine's priority needs on the battlefield. NATO allies then locate the weapons and ammunition and send them on. 'Packages will be prepared rapidly and issued on a regular basis,' NATO said Monday. Air defense systems are in greatest need. The United Nations has said that Russia's relentless pounding of urban areas behind the front line has killed more than 12,000 Ukrainian civilians. Russia's bigger army is also making slow but costly progress along the 1,000-kilometer (620-mile) front line. Currently, it is waging an operation to take the eastern city of Pokrovsk, a logistical hub whose fall could allow it to drive deeper into Ukraine. European allies and Canada are buying most of the equipment they plan to send from the United States, which has greater stocks of ready military materiel, as well as more effective weapons. The Trump administration is not giving any arms to Ukraine. The new deliveries will come on top of other pledges of military equipment. The Kiel Institute, which tracks support to Ukraine, estimates that as of June, European countries had provided 72 billion euros ($83 billion) worth of military aid since the start of Russia's full-scale invasion in February 2022, compared to $65 billion in U.S. aid. Dutch Defense Minister Ruben Brekelmans said that 'American air defense systems and munitions, in particular, are crucial for Ukraine to defend itself.' Announcing the deliveries Monday, he said Russia's attacks are 'pure terror, intended to break Ukraine.' President Volodymyr Zelenskyy expressed his gratitude to the Netherlands, posting on social media that 'Ukraine, and thus the whole of Europe, will be better protected from Russian terror.' He said the deliveries are coming 'at a time when Russia is trying to scale up its strikes. This will definitely help protect the lives of our people!' Germany said Friday it will deliver two more Patriot air defense systems to Ukraine in the coming days. It agreed to the move after securing assurances that the U.S. will prioritize the delivery of new Patriots to Germany to backfill its stocks. These weapon systems are only made in the U.S. As an organization, NATO provides only non-lethal assistance to Ukraine like uniforms, tents, medical supplies and logistics support. The 32-nation military alliance has mostly sought to protect NATO territory from possible Russian attack and avoid being dragged into a war against a nuclear power. But its support role has expanded since U.S. President Donald Trump took office in January, even as his administration insists European allies must now take care of their own security and that of their war-ravaged neighbor. Trump has made no public promise of weapons or economic support for Ukraine. Trump said on July 28 that the U.S. is "going to be sending now military equipment and other equipment to NATO, and they'll be doing what they want, but I guess it's for the most part working with Ukraine.' ___ Mike Corder in The Hague, Netherlands, and Kirsten Grieshaber and David Keyton in Berlin contributed to this report. Lorne Cook, The Associated Press
New York Post
3 minutes ago
- New York Post
Ditch the razor and get 21% off the Braun smart IPL with 4K already sold
New York Post may be compensated and/or receive an affiliate commission if you click or buy through our links. Featured pricing is subject to change. Summer's not over yet, and now that your legs have a little glow, it's the perfect time to get smoother skin while you're rocking those short shorts. If you're tired of the constant cycle of shaving, waxing, or booking expensive salon laser appointments, the Braun Silk-Expert Pro 5 might be the home solution you've been looking for. Reduce cuts on your legs from hastily shaving before work. Think no more cuts, just smooth-looking legs. This FDA-cleared IPL (intense pulsed light) device promises up to 97% hair reduction in just four to twelve weeks when used consistently. Advertisement It uses smart SensoAdapt technology to automatically adjust the intensity based on your skin tone for a safe, effective treatment every time. With up to 400,000 flashes, it's designed to last for years, and treat your full body in minutes thanks to its high-speed flash rate. If you're searching for the best fit, check out our review of the top IPL hair removal devices on the market. Ready for immaculate, hair-free legs? Join the beauty club at a discount with Braun. Amazon It's pain-free, without yanking or waxing strips. Just a quick, gentle flash of light with a precision head for trickier areas like your face, underarms, and bikini line, so you can go full spa-mode at home. That's right, you don't have to worry about those awkward, one-leg-up poses at the salon. For over 200 years, the New York Post has been America's go-to source for bold news, engaging stories, in-depth reporting, and now, insightful shopping guidance. We're not just thorough reporters – we sift through mountains of information, test and compare products, and consult experts on any topics we aren't already schooled specialists in to deliver useful, realistic product recommendations based on our extensive and hands-on analysis. Here at The Post, we're known for being brutally honest – we clearly label partnership content, and whether we receive anything from affiliate links, so you always know where we stand. We routinely update content to reflect current research and expert advice, provide context (and wit) and ensure our links work. Please note that deals can expire, and all prices are subject to change.
Chicago Tribune
3 minutes ago
- Chicago Tribune
The world nearly beat polio. But fake records, an imperfect vaccine and missteps aided its comeback
KARACHI, Pakistan — For the past decade, Sughra Ayaz has traveled door to door in southeastern Pakistan, pleading with parents to allow children to be vaccinated against polio as part of a global campaign to wipe out the paralytic disease. She hears their demands and fears. Some are practical – families need basics like food and water more than vaccines. Others are simply unfounded – the oral doses are meant to sterilize their kids. Amid rampant misinformation and immense pressure for the campaign to succeed, Ayaz said, some managers have instructed workers to falsely mark children as immunized. And the vaccines, which must be kept cold, aren't always stored correctly, she added. 'In many places, our work is not done with honesty,' Ayaz said. The World Health Organization and partners embarked on their polio campaign in 1988 with the bold goal of eradication — a feat seen only once for human diseases, with smallpox in 1980. They came close several times, including in 2021, when just five cases of the natural virus were reported in Pakistan and Afghanistan. But since then, cases rebounded, hitting 99 last year, and officials have missed at least six self-imposed eradication deadlines. Afghanistan and Pakistan remain the only countries where transmission of polio — which is highly infectious, affects mainly children under 5, and can cause irreversible paralysis within hours — has never been interrupted. The worldwide campaign has focused most of its attention and funding there for the past decade. But in its quest to eliminate the disease, the Global Polio Eradication Initiative has been derailed by mismanagement and what insiders describe as blind allegiance to an outdated strategy and a problematic oral vaccine, according to workers, polio experts and internal materials obtained by The Associated Press. Officials have falsified vaccination records, selected unqualified people to dole out drops, failed to send out teams during mass campaigns, and dismissed concerns about the oral vaccine sparking outbreaks, according to documents shared with AP by staffers from GPEI – one of the largest and most expensive public health campaigns in history, with over $20 billion spent and nearly every country in the world involved. In Afghanistan and Pakistan – which share a border, harbor widespread mistrust of vaccines, and have weakened healthcare systems and infrastructure – local staffers like Ayaz have for years flagged problems to senior managers. But those issues, along with concerns by staffers and outside health officials, have long gone unaddressed, insiders say. Officials tout the successes – 3 billion children vaccinated, an estimated 20 million people who would have been paralyzed spared – while acknowledging challenges in Pakistan and Afghanistan. Remote villages are hard to reach, some cultural and religious authorities instruct against vaccination, and hundreds of polio workers and security staff have been killed because of their alignment with a Western-led initiative. Dr. Jamal Ahmed, WHO's polio director, defended progress in those two countries, citing workers' tailored response in resistant pockets. 'There's so many children being protected today because of the work that was done over the past 40 years,' he said. 'Let's not overdramatize the challenges, because that leads to children getting paralyzed.' Ahmed said he believes authorities will end the spread of polio in the next 12 to 18 months. Its latest goal for eradication is 2029. The campaign says about 45 million children in Pakistan and 11 million in Afghanistan must be vaccinated this year. Children typically need four doses of two drops each to be considered fully immunized. Dr. Zulfiqar Bhutta, who has served on advisory groups for WHO, the Gates Foundation and others, said campaign officials should listen to the criticism of its tactics. 'Continuing blindly with the same strategies that we have relied on since eradication began is unlikely to lead to a different result,' he said. Internal WHO reports reviewing vaccination drives in Afghanistan and Pakistan over the past decade – given to AP by current and former staffers – show that as early as 2017, local workers were alerting significant problems to senior managers. The documents flagged multiple cases of falsified vaccination records, health workers being replaced by untrained relatives and workers improperly administering vaccines. On numerous occasions, WHO officials noted, 'vaccinators did not know about vaccine management,' citing failure to keep doses properly cold. They also found sloppy or falsified reporting, with workers noting 'more used vaccine vials than were actually supplied.' According to an August 2017 report from Kandahar, Afghanistan, local government authorities and others interfered in choosing vaccinators, 'resulting in the selection of underage and illiterate volunteers.' Vaccination teams worked 'in a hurried manner,' reports said, with 'no plan for monitoring or supervision.' A team in Nawzad, Afghanistan, covered just half of the intended area in 2017, with 250 households missed entirely. Village elders said no one visited for at least two years. Vaccine workers and health officials in Afghanistan and Pakistan confirmed the issues in the documents and told AP it's hard for campaign leadership to grasp the difficulties in the field. Door-to-door efforts are stymied by cultural barriers, unfounded stories about vaccines, and the region's poverty and transience. 'Most of the time when we go to vaccinate and knock on the door, the head of the house or the man is not at home,' said one worker, speaking on condition of anonymity because they weren't authorized to talk to the press. 'Many people find it offensive that a stranger knocks on the door and talks to a woman.' Some workers find families have moved. Occasionally, they say, the encounter abuse. 'We have shared these problems with our senior officials,' the worker told AP. 'They know about it.' In an email response to AP's questions about officials' knowledge of the issues, WHO polio director Ahmed noted 'operational challenges' in Afghanistan and Pakistan and said the program has 'robust monitoring and evaluation processes.' Worker Ayaz described 'fake finger marking' — placing the ink used to show a child is vaccinated on their pinky even when no vaccine has been given. 'There is so much pressure,' Ayaz said. Before the first polio vaccine was developed in 1955, the disease — spread mostly from person to person, through contaminated water and via fecal particles — was among the world's most feared, paralyzing hundreds of thousands of children annually. People avoided crowded places during epidemics, and hospital wards filled with children encased in iron lungs after the virus immobilized their breathing muscles. Polio is mainly spread when people are exposed to water infected with the virus. In countries with poor sanitation, children often become infected when they come into contact with contaminated waste. WHO says that as long as a single child remains infected, kids everywhere are at risk. Eradication demands near-perfection – zero polio cases and immunizing more than 95% of children. But public health leaders and former WHO staffers say campaign efforts are far from perfect, and many question the oral vaccine. The oral vaccine – proven to be safe and effective — has been given to more than 3 billion children. But there are some extremely rare side effects: Scientists estimate that for every 2.7 million first doses given, one child will be paralyzed by the live polio virus in the vaccine. In even rarer instances, the live virus can mutate into a form capable of starting new outbreaks among unimmunized people where vaccination rates are low. Worldwide, several hundred vaccine-derived cases have been reported annually since at least 2021, with at least 98 this year. Most public health experts agree the oral vaccine should be pulled as soon as possible. But they acknowledge there simply isn't enough injectable vaccine — which uses no live virus and doesn't come with the risks of the oral vaccine — to wipe out polio alone. The injectable vaccine also is more expensive and requires more training to administer. More than two dozen current and former senior polio officials told AP the agencies involved haven't been willing to even consider revising their strategy to account for some of the campaign's problems. Dr. Tom Frieden, a former director of the U.S. Centers for Disease Control and Prevention who sits on an independent board reviewing polio eradication, said it would be impossible to eliminate polio without the oral vaccine. But he's urged authorities to find ways to adapt, such as adopting new methods to identify polio cases more quickly. Since 2011, he and colleagues have issued regular reports about overall program failures. 'There's no management,' he said, citing a lack of accountability. Last year, former WHO scientist Dr. T. Jacob John twice emailed WHO Director-General Tedros Adhanom Ghebreyesus calling for a 'major course correction.' John shared the emails with AP and said he's received no response. 'WHO is persisting with polio control and creating polio with one hand and attempting to control it by the other,' John wrote. In his response to AP, WHO polio director Ahmed said the oral vaccine is a 'core pillar' of eradication strategy and that 'almost every country that is polio-free today used (it) to achieve that milestone.' 'We need to step back and really care for the people,' he said. 'The only way we can do that in large parts of the world is with oral polio vaccine.' Ahmed also pointed to the success WHO and partners had eliminating polio from India, once considered a nearly impossible task. In the four years before polio was wiped out there, health workers delivered about 1 billion doses of the oral vaccine to more than 170 million children. Today, nearly all of the world's polio cases — mostly in Africa and the Middle East — are mutated viruses from the oral vaccine, except for Afghanistan and Pakistan. Scott Barrett, a Columbia University professor, called for an inquiry into how things went so wrong – particularly with a failed effort in 2016, when authorities removed a strain from the oral vaccine. They miscalculated, leading to outbreaks in more than 40 countries that paralyzed more than 3,000 children, according to an expert report commissioned by WHO. Last year, a mutated virus traced to that effort paralyzed a baby in Gaza. 'Unless you have a public inquiry where all the evidence comes out and WHO makes serious changes, it will be very hard to trust them,' he said. With an annual budget of about $1 billion, the polio initiative is among the most expensive in all of public health. This year, the U.S. withdrew from WHO, and President Donald Trump has cut foreign aid. WHO officials have privately admitted that sustaining funding would be difficult without success. Some say the money would be better spent on other health needs. 'We have spent more than $1 billion (in external polio funding) in the last five years in Pakistan alone, and it didn't buy us any progress,' said Roland Sutter, who formerly led polio research at WHO. 'If this was a private company, we would demand results.' Villagers, too, have protested the cost, staging hundreds of boycotts of immunization campaigns since 2023. Instead of polio vaccines, they ask for medicine, food and electricity. In Karachi, locals told AP they didn't understand the government's fixation on polio and complained of other issues — dirty water, heroin addiction. Workers are accompanied by armed guards; Pakistani authorities say more than 200 workers and police assigned to protect them have been killed since the 1990s, mostly by militants. The campaign also is up against a wave of misinformation, including that the vaccine is made from pig urine or will make children reach puberty early. Some blame an anti-vaccine sentiment growing in the U.S. and other countries that have largely funded eradication efforts and say it's reaching even remote areas of Afghanistan and Pakistan. In suburban southwest Pakistan, Saleem Khan, 58, said two grandchildren under 5 were vaccinated over his family's objections. 'It results in disability,' said Khan, without citing evidence for his belief. 'They are vaccinated because officials reported our refusal to authorities and the police.' Svea Closser, professor of international health at Johns Hopkins University, said Pakistan and Afghanistan were less resistant to immunization decades ago. Now, people are angry about the focus on polio and lack of help for diseases like measles or tuberculosis, she said, spurring conspiracy theories. 'Polio eradication has created a monster,' Closser said. It doesn't help, she added, that in this region, public trust in vaccine campaigns was undermined when the CIA organized a fake hepatitis drive in 2011 in an attempt to get DNA and confirm the presence of Osama bin Laden or his family. Workers see that continued mistrust every day. In a mountainous region of southeastern Afghanistan where most people survive by growing wheat and raising cows and chickens, a mother of five said she'd prefer that her children be vaccinated against polio, but her husband and other male relatives have instructed their families to reject it. They believe the false rumors that it will compromise their children's fertility. 'If I allow it,' the woman said, declining to be named over fears of family retribution, 'I will be beaten and thrown out.'
