FDA CDC News
FDA Approves First Treatment for Acid Sphingomyelinase Deficiency, a Rare Genetic Disease
Today, the U.S. Food and Drug Administration approved Xenpozyme (Olipudase alfa) for intravenous infusion in pediatric and adult patients with Acid Sphingomyelinase Deficiency (ASMD), a rare genetic disease that causes premature death. Xenpozyme is the first approved medication to treat symptoms that are not related to the central nervous system in patients with ASMD.
“ASMD has a debilitating effect on people’s lives and there is a critical need to increase treatment options for patients who suffer from this rare disease,” said Christine Nguyen, M.D., deputy director of the Office of Rare Diseases, Pediatrics, Urologic and Reproductive Medicine in the FDA’s Center for Drug Evaluation and Research. “The challenges involved with developing treatments for rare diseases are significant and unique. We believe patients who suffer from ASMD, their families and their physicians will welcome this long-awaited advancement.”
ASMD is caused by the lack of an enzyme needed to break down a complex lipid, called sphingomyelin, that accumulates in the liver, spleen, lung, and brain. Patients with ASMD have enlarged abdomens that can cause pain, vomiting, feeding difficulties, and falls. They also have abnormal liver and blood tests. The most severely affected patients have profound neurologic symptoms and rarely survive beyond two to three years of age. Other patients may survive into adulthood but die prematurely from respiratory failure.
Xenpozyme is an enzyme replacement therapy that helps reduce sphingomyelin accumulation in the liver, spleen, and lung. The efficacy of Xenpozyme for the treatment of ASMD was demonstrated in a randomized, double-blind, placebo-controlled study of 31 patients randomized to take Xenpozyme or placebo. Because the study had the placebo comparator and measured treatment benefits that could be detected during the study’s duration, the FDA was able to conclude that Xenpozyme is effective. Overall, treatment with Xenpozyme improved lung function and reduced liver and spleen size.
The most common side effects of Xenpozyme include headache, cough, fever, joint pain, diarrhea, and low blood pressure. Xenpozyme carries a boxed warning for severe hypersensitivity reactions including anaphylaxis. Some patients treated with Xenpozyme developed laboratory test abnormalities, such as abnormal liver blood tests. Routine blood laboratory testing should be obtained periodically. Xenpozyme should not be started during pregnancy due to the potential for fetal harm, which was observed during animal studies. Additionally, in the clinical trials, 75% of pediatric patients and 50% of adult patients experienced reactions including headaches, nausea and vomiting while receiving Xenpozyme through intravenous infusion.
Xenpozyme received fast track, breakthrough therapy, and priority review designations. It also received orphan drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases. The FDA awarded the sponsor a rare pediatric disease priority review voucher, an incentive to encourage development of new drugs and biologics that prevent and treat rare diseases in children.
More than 7,000 rare diseases affect more than 30 million people in the United States. Many rare conditions are life threatening and most do not have treatments. The FDA estimates that half of these serious or life-threatening diseases affect children.
The FDA granted the approval of Xenpozyme to Genzyme.
Related Information
- Office of Rare Diseases, Pediatrics, Urologic and Reproductive Medicine-Division of Rare Diseases and Medical Genetics (DRDMG)
- Medical products for rare diseases and conditions
- CDER’s ARC Program
Source: FDA
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FDA CDC News
Seafood Recall: Important Information on Hannaford Seafood Salads
In early November, the U.S. Food and Drug Administration (FDA) announced a Class II recall concerning Hannaford Seafood Salads, affecting over 1,600 units across five states: Maine, New Hampshire, Vermont, Massachusetts, and New York. The recall, initiated on October 31, was due to a mislabeling incident that poses a significant risk to consumers with soy allergies.
Understanding the Class II Risk Level
The FDA has classified this recall as a Class II risk, indicating that exposure to the affected products may cause “temporary or medically reversible adverse health consequences.” However, the risk of experiencing serious health consequences remains “remote.” Despite this classification, consumers with an allergy or severe sensitivity to soy must take this recall seriously, as they could face life-threatening allergic reactions if they consume the mislabeled salads.
What Happened?
The problem arose when the printer responsible for the salad labels provided Elevation Foods, the manufacturer of the salads, with labels that did not accurately reflect the current product formulation. Instead of the correct labels, the printer mistakenly issued labels meant for an upcoming formulation change. As a result, the affected salads may contain undeclared hydrolyzed soy protein, a known allergen that must be clearly stated on product labels to protect consumer safety.
Product Details
The implicated Hannaford Seafood Salads were distributed between October 25 and 29, and they feature use-by dates of November 13 and 14. Consumers can find these dates printed on the lid of the containers. Following the recall announcement, all affected products were promptly removed from store shelves.
Health Risks Associated with Soy Allergies
For individuals with soy allergies, the immune system mistakenly identifies soy proteins as harmful invaders, leading to a range of symptoms that can vary in severity. Common reactions include stomach issues, coughing, itching, wheezing, vomiting, hives, and difficulty breathing. In severe cases, these reactions can escalate to life-threatening conditions, such as anaphylaxis.
To mitigate risks, those with soy allergies are advised to carry an epinephrine injection (Epi-Pen) to counteract severe allergic reactions swiftly.
What Should Consumers Do?
Customers who purchased Hannaford Seafood Salads before the recall are urged not to consume the products. Instead, they can return them to the store where they made the purchase for a full refund. Elevation Foods has not reported any known cases of illness related to these salads, but consumer safety remains a top priority.
Conclusion
This seafood salad recall serves as a crucial reminder about the importance of accurate labeling, especially when it comes to potential allergens. Consumers should remain vigilant and informed about the products they purchase, particularly if they have food allergies. Stay safe and always check your labels!
Related Links:
https://www.newsweek.com/seafood-salad-recall-update-fda-risk-level-hannaford-1992294
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pets
Urgent Dog Treat Recall: Carolina Prime Pet Issues Warning Due to Salmonella Risk
Dog Treat Recall
In a recent announcement that has concerned dog owners across the Southeastern, Midwestern, and Mid-Atlantic regions of the United States, the FDA has reported a recall of “Hollywood Feed Carolina Made Chicken Chips” dog treats manufactured by Carolina Prime Pet, Inc. of Lenoir, North Carolina. This recall, affecting approximately 400 bags of these popular dog treats, is attributed to the potential risk of Salmonella contamination.
Why Is This Recall Important?
While there have been no reported illnesses linked to the consumption of these chicken chips, the safety of pets—and by extension, their human companions—is paramount. Salmonella, a bacteria that can cause severe gastrointestinal distress in animals and humans alike, can be particularly dangerous for pets, leading to symptoms such as vomiting, diarrhea, and lethargy. For individuals, Salmonella can also result in serious health complications, especially in young children, elderly adults, or those with weakened immune systems.
Details of the Recall
The recall specifically pertains to 16-ounce bags of “Hollywood Feed Carolina Made Chicken Chips” that carry the lot number 20051324L2 and an expiration date of 11/13/25. Consumers can easily identify the affected product by checking the back of the packaging, which features a distinctive purple-and-black design. The recall extends to retail locations across 19 states, including:
- Alabama
- Arkansas
- Florida
- Georgia
- Illinois
- Indiana
- Kansas
- Kentucky
- Louisiana
- Michigan
- Mississippi
- Missouri
- North Carolina
- Ohio
- Oklahoma
- Pennsylvania
- South Carolina
- Tennessee
- Texas
For those who purchased the treats online, they were also available on the company’s website at www.hollywoodfeed.com.
What Should Consumers Do?
Carolina Prime Pet, Inc. has advised consumers to check their homes for the affected product. If your dog treats match the recalled lot number and expiration date, it is crucial not to feed them to your pet. Instead, return the products to the place of purchase for a full refund. For those who have questions or need more information, the company encourages contacting their customer service.
Final Thoughts
As pet owners, it is our responsibility to stay informed about the products we give our furry friends. The safety of our pets is essential, and recalls like these serve as a reminder to always check product packaging and stay updated on food safety announcements. If you have any concerns about your pet’s health or have noticed any unusual symptoms after consuming potentially contaminated food, it is advisable to consult with a veterinarian immediately.
Stay alert, stay informed, and together we can ensure the safety and well-being of our beloved pets.
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FDA CDC News
Popular Nasal Decongestant Falls Short: FDA Advisers Question Efficacy
FDA questions effectiveness of popular nasal decongestant phenylephrine, raising concerns about congestion relief.
In a surprising turn of events, government experts have cast doubt on the effectiveness of the leading decongestant found in popular over-the-counter medications like Sudafed and Dayquil. A recent review conducted by the Food and Drug Administration (FDA) found that the main drug ingredient, phenylephrine, offers no significant relief from nasal congestion. This revelation raises concerns among millions of Americans who rely on these medications for congestion relief.
Phenylephrine became the primary ingredient in over-the-counter decongestants after pseudoephedrine, an older ingredient, was moved behind pharmacy counters due to its potential for illegal drug processing. However, the FDA’s outside advisers unanimously voted against the efficacy of phenylephrine, prompting a reevaluation of its effectiveness. Despite phenylephrine-based versions dominating the market, accounting for four-fifths of the $2.2 billion oral decongestant market, the FDA’s conclusions suggest a need to reconsider their availability.
The FDA review identified significant flaws in the original studies that supported phenylephrine’s approval. These studies, conducted in the 1960s and 1970s, were deemed inadequate by modern standards. Furthermore, three recent, well-conducted studies by reputable pharmaceutical companies found no discernible difference between phenylephrine medications and placebos in relieving congestion. This raises concerns about the effectiveness of phenylephrine, which has relied on outdated research.
The FDA’s advisory panel’s unanimous vote against phenylephrine’s effectiveness could lead to regulatory action requiring drugmakers like Johnson & Johnson and Bayer to withdraw their phenylephrine-based oral decongestants from store shelves. This move may force consumers to opt for behind-the-counter pseudoephedrine products or phenylephrine-based nasal sprays and drops. However, it would necessitate extensive education efforts by the FDA, pharmacists, and drugstores to inform consumers about alternative options for treating congestion.
The Consumer Healthcare Products Association, representing nonprescription drugmakers, argued that the recent studies have limitations and advocated for continued “easy access” to phenylephrine. However, the FDA review highlighted the shortcomings of the original studies and the lack of modern scientific evidence supporting phenylephrine’s efficacy. Balancing public health concerns and consumer access will be a crucial challenge for regulators moving forward.
The FDA’s recent review has cast doubt on the effectiveness of phenylephrine, the key ingredient in popular nasal decongestants. This finding prompts a reevaluation of treatment options for nasal congestion and calls for further research to provide effective relief for individuals in need.
Check out the article from the Associated Press: https://apnews.com/article/sudafed-decongestants-phenylephrine-pseudoephedrine-fda-0f140bafae9a500c5fba05fe764ecb66
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