FDA Says Sarepta Can Treat Ambulatory Patients With Gene Therapy
In a statement Monday, the regulator said that it had concluded that the death of an 8-year-old in Brazil was not related to the therapy.
Hashtags
Try Our AI Features
Explore what Daily8 AI can do for you:
Comments
No comments yet...
Related Articles
Yahoo
23 minutes ago
- Yahoo
Novo Nordisk outgoing CEO blames compounded GLP-1s for decline in 2025 outlook
Novo Nordisk's (NVO) downward guidance Tuesday sent its stock diving 20%, even as new leadership remains bullish on the company's ability to maintain a market lead in the obesity drug space. The maker of GLP-1 drugs Ozempic and Wegovy cut sales grown from 13%-21% down to 8%-14% for the year. That's about half, on the higher end, of the 26% growth seen in 2024. Outgoing CEO Lars Jøregensen told reporters Tuesday that the compounding, or copycat, market in the US is to blame. That is one of two key issues that appear to have taken the company by surprise. First was the shortage of both its GLP-1 drugs after they unexpectedly zoomed to popularity, creating a new weight-loss drug market. Jørgensen said that the prevalence of compounded products, which are now restricted after the FDA declared an end to the shortage, is to blame. "Despite the fact that it is now illegal ... it's still happening. And that's one of the assumptions in the prior guidance, that that would be reduced significantly. And we see, actually, that is actually the same volume as we saw earlier in the year, around a million patients using a compounded product," Jørgensen said. The compounded products include those from Novo's ex-partner, Hims & Hers (HIMS), which continues to sell the products based on a loophole in FDA rules that allows it to sell "personalized" products for patients who might have negative reactions or side effects to branded drugs — such as common GLP-1 side effects like nausea or dizziness. Novo recently ended its direct-to-consumer sales deal with Hims after the telehealth firm declined to stop selling compounded semaglutide vials. The result has been a loss in the company's first-to-market position, with competitor Eli Lilly (LLY) taking a harder stance, most recently lobbying Congress to help enforce the FDA rules and rid the market of compounding. Jørgensen said Tuesday, "Ultimately, compounding will disappear. But it will take some time before it's completely out of the market and we have a fair chance of regaining our fair share (of the market)." Incoming CEO Mike Doustdar, who has served as Novo's executive vice president of international operations, said the decline of the company's stock, and therefore shareholder value, is disappointing. "I don't like it as an employee ... as a CEO-elect, and I certainly don't like it as a shareholder myself. But setbacks don't define companies, our response does," Doustdar said, adding that the guidance cut reinforces his mandate to put the company back onto a growth path. Anjalee Khemlani is the senior health reporter at Yahoo Finance, covering all things pharma, insurance, provider services, digital health, PBMs, and health policy and politics. That includes GLP-1s, of course. Follow Anjalee as AnjKhem on social media platforms X, LinkedIn, and Bluesky @AnjKhem. Click here for in-depth analysis of the latest health industry news and events impacting stock prices
Hamilton Spectator
an hour ago
- Hamilton Spectator
US health officials crack down on kratom-related products after complaints from supplement industry
WASHINGTON (AP) — U.S. health officials are warning Americans about the risks of an opioid-like ingredient increasingly added to energy drinks, gummies and supplements sold at gas stations and convenience stores, recommending a nationwide ban. The chemical, known as 7- hydroxymitragynine , is a component of kratom , a plant native to Southeast Asia that has gained popularity in the U.S. as an unapproved treatment for pain, anxiety and drug dependence. In recent months, dietary supplement companies that sell kratom have been urging the Food and Drug Administration to crack down on the products containing 7-OH, portraying them as dangerously strong, synthetic versions of the original ingredient. The FDA action 'is not focused on natural kratom leaf products,' according to a statement Tuesday by the U.S. Department of Health and Human Services. The agency said it was releasing a report to educate about the risks of '7-OH and its distinction from the kratom plant leaf.' Regulators are also recommending that the ingredient be placed on the federal government's most restrictive list of illegal drugs, which also includes LSD and heroin. '7-OH is an opioid that can be more potent than morphine,' said FDA Commissioner Marty Makary. 'We need regulation and public education to prevent another wave of the opioid epidemic.' The agency's recommendation will be reviewed by the Drug Enforcement Administration, which sets federal rules for high-risk drugs including prescription medicines and illicit substances. Federal regulators have been scrutinizing kratom for about a decade after reports of addiction, injury and overdose. But users and distributors have long opposed efforts to regulate it, saying kratom could be a safer alternative to opioid painkillers that sparked the ongoing drug addiction epidemic. Last month, the FDA issued warning letters to seven companies selling drinks, gummies and powders infused with the drug. Regulators said the products violated FDA rules because they have not been evaluated for safety and, in some cases, claimed to treat medical conditions, including pain, arthritis and anxiety. Supplement executives quickly applauded the move. The FDA 'demonstrated the exact kind of data-driven, proactive regulatory excellence needed to safeguard unwitting consumers across the U.S.,' said Ryan Niddel of Diversified Botanics, a Utah-based company that sells kratom supplements. An industry group, the American Kratom Association, has lobbied Congress for years against restrictions on the plant. Legislation supported by the group would prohibit the FDA from regulating kratom more strictly than food and dietary supplements Nearly a decade ago, the federal government came close to a national ban on the substance. In 2016, the DEA announced plans to add kratom to the government's most restrictive schedule 1 of illegal drugs. But the plan stalled after a flood of public complaints, including a letter signed by more than 60 members of Congress. The FDA then began studying the ingredient, concluding in 2018 that kratom contains many of the same chemicals as opioids, the addictive class of drugs that includes painkillers like OxyContin as well as heroin and fentanyl. Since then, FDA regulators have continued to issue warnings about cases of injury, addiction and death with kratom supplements, which are usually sold in capsules or powders. In recent months, the FDA has also issued warnings on other unapproved drugs sold as supplements or energy drinks, including the antidepressant tianeptine . Sometimes referred to collectively as 'gas station heroin,' the drugs have been restricted by several states, but they are not scheduled at the federal level. ___ 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.
Associated Press
an hour ago
- Associated Press
US health officials crack down on kratom-related products after complaints from supplement industry
WASHINGTON (AP) — U.S. health officials are warning Americans about the risks of an opioid-like ingredient increasingly added to energy drinks, gummies and supplements sold at gas stations and convenience stores, recommending a nationwide ban. The chemical, known as 7- hydroxymitragynine, is a component of kratom, a plant native to Southeast Asia that has gained popularity in the U.S. as an unapproved treatment for pain, anxiety and drug dependence. In recent months, dietary supplement companies that sell kratom have been urging the Food and Drug Administration to crack down on the products containing 7-OH, portraying them as dangerously strong, synthetic versions of the original ingredient. The FDA action 'is not focused on natural kratom leaf products,' according to a statement Tuesday by the U.S. Department of Health and Human Services. The agency said it was releasing a report to educate about the risks of '7-OH and its distinction from the kratom plant leaf.' Regulators are also recommending that the ingredient be placed on the federal government's most restrictive list of illegal drugs, which also includes LSD and heroin. '7-OH is an opioid that can be more potent than morphine,' said FDA Commissioner Marty Makary. 'We need regulation and public education to prevent another wave of the opioid epidemic.' The agency's recommendation will be reviewed by the Drug Enforcement Administration, which sets federal rules for high-risk drugs including prescription medicines and illicit substances. Federal regulators have been scrutinizing kratom for about a decade after reports of addiction, injury and overdose. But users and distributors have long opposed efforts to regulate it, saying kratom could be a safer alternative to opioid painkillers that sparked the ongoing drug addiction epidemic. Last month, the FDA issued warning letters to seven companies selling drinks, gummies and powders infused with the drug. Regulators said the products violated FDA rules because they have not been evaluated for safety and, in some cases, claimed to treat medical conditions, including pain, arthritis and anxiety. Supplement executives quickly applauded the move. The FDA 'demonstrated the exact kind of data-driven, proactive regulatory excellence needed to safeguard unwitting consumers across the U.S.,' said Ryan Niddel of Diversified Botanics, a Utah-based company that sells kratom supplements. An industry group, the American Kratom Association, has lobbied Congress for years against restrictions on the plant. Legislation supported by the group would prohibit the FDA from regulating kratom more strictly than food and dietary supplements Nearly a decade ago, the federal government came close to a national ban on the substance. In 2016, the DEA announced plans to add kratom to the government's most restrictive schedule 1 of illegal drugs. But the plan stalled after a flood of public complaints, including a letter signed by more than 60 members of Congress. The FDA then began studying the ingredient, concluding in 2018 that kratom contains many of the same chemicals as opioids, the addictive class of drugs that includes painkillers like OxyContin as well as heroin and fentanyl. Since then, FDA regulators have continued to issue warnings about cases of injury, addiction and death with kratom supplements, which are usually sold in capsules or powders. In recent months, the FDA has also issued warnings on other unapproved drugs sold as supplements or energy drinks, including the antidepressant tianeptine. Sometimes referred to collectively as 'gas station heroin,' the drugs have been restricted by several states, but they are not scheduled at the federal level. ___ 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.
