Ultragenyx receives CRL from FDA for UX111 gene therapy
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Yahoo
24 minutes ago
- Yahoo
US FDA approves Ionis' drug for rare genetic disorder
By Sneha S K and Bageshri Banerjee (Reuters) -The U.S. Food and Drug Administration on Thursday approved Ionis Pharmaceuticals' drug to prevent instances of severe swelling in various parts of the body of patients with a rare genetic disorder. Shares of the company were up 1.1% in afternoon trading. The drug, Dawnzera, is approved as a so-called "prophylaxis" to prevent symptoms of a rare genetic disease called hereditary angioedema (HAE) in adults and pediatric patients 12 years of age and older. The condition causes frequent attacks of severe swelling in various parts of the body, including the hands, feet, genitals and face. Dawnzera will be available in the United States in the coming days with a list price of $57,462 per dose, the company said. HAE is estimated to affect about 7,000 patients in the U.S., according to Ionis. The drug, chemically known as donidalorsen, prevents HAE attacks by lowering levels of a protein called prekallikrein (PKK) that drives swelling and inflammation. Dawnzera can be self-administered through an under-the-skin injection once every four or eight weeks. The approval was supported by late-stage study results that showed the drug, dosed once every four weeks, reduced monthly attacks by 81% compared to a placebo over 24 weeks. Current FDA-approved therapies for HAE fall into two groups: prophylaxis to prevent attacks and on-demand medicines to treat acute attacks. Other approved prophylactic drugs include Takhzyro, developed by Japan's Takeda Pharmaceutical and Haegarda from CSL Behring. There is a lot of excitement for donidalorsen as patients who are not doing well on existing therapies have an option to switch to Dawnzera, which has a higher efficacy, TD Cowen analyst Yaron Werber said ahead of the approval. Takhzyro, given every two weeks, showed an average of 87% fewer attacks compared to a placebo over 6.5 months in a separate study involving adult and adolescent patients. Werber estimates peak annual sales of Ionis' drug to be $509 million by 2032. Solve the daily Crossword
USA Today
2 hours ago
- USA Today
Was the potentially radioactive shrimp sold in your state? See map.
A recall has been issued for frozen shrimp sold at Walmart in 13 states that may have been contaminated with a radioactive chemical, the Food and Drug Administration said. On Tuesday, Aug. 19, the FDA advised the recall after it detected Cesium-137 (Cs-137), a radioactive chemical "produced by nuclear fission for use in medical devices and gauges," according to the Environmental Protection Agency. The chemical was detected in four U.S. ports across the country and in a sample of breaded shrimp, the FDA stated. "Although testing to date has not confirmed the presence of contamination in any product in commerce, the product appears to have been prepared, packed, or held under insanitary conditions whereby it may have become contaminated with Cs-137 and may pose a safety concern," the FDA advised. The FDA is investigating the situation. Which states sold the recalled frozen shrimp? The recalled shrimp were sold in the following 13 states: More recalls: Over 78,000 Jeep Wrangler vehicles recalled. Here's why. Ports that received contaminated shipping containers The four ports where the FDA detected contaminated shipping containers are in the following cities: Which products were recalled? The recall is targeted at 2-pound bags of Great Value brand Frozen Raw Ez Peel Tail-On Farm-Raised White Vannamei Shrimp. Lot codes and best by dates include the following: The shrimp products are processed by PT. Bahari Makmur Sejati, a company in Indonesia doing business as BMS Foods, according to the FDA. What to do if you have recalled shrimp If you have a package of the recalled Walmart shrimp at home, throw it away and do not eat or serve it, the FDA notice says. Distributors and retailers should dispose of the product and refrain from selling it. Anybody who fears they may have been exposed to elevated levels of cesium should contact their medical providers. To submit a complaint or report an adverse reaction, consumers can visit the FDA's Industry and Consumer Assistance portal. PT. Bahari Makmur Sejati placed under an import alert PT. Bahari Makmur Sejati has also been placed on an import alert for chemical contamination, which prevents any of the company's food products from entering the United States until it resolves any conditions that could have caused the exposure. The FDA is working with Indonesian seafood regulatory authorities to investigate the cause of the contamination. What is Cesium-137 (Cs-137) and why is it regulated? Cs-137 is a radioisotope of cesium, meaning it is a chemical element that emits radiation as it breaks down. It is man-made and is produced by nuclear fission, according to the FDA. In the United States, it is used in medical devices and measurement gauges, such as industrial devices that measure the thickness of materials. "It is also one of the byproducts of nuclear fission processes in nuclear reactors and nuclear weapons testing," the EPA stated. Because it is widespread around the globe, trace amounts can be found in the environment, including in soil, food and air, the FDA. Agencies, including the FDA and U.S. Customs & Border Protection, test for, monitor and regulate the presence of the substance due to the risks associated with long-term exposure. Low-level radiation exposure over time can lead to serious health complications, the FDA said. Exposure to Cs-137 alone can cause burns, acute radiation sickness, cancer and death. As a result, the governing agencies restrict potential exposure to lessen the possibility of these long-term impacts. Julia is a trending reporter for USA TODAY. Connect with her on LinkedIn, X, Instagram and TikTok: @juliamariegz, or email her at jgomez@
Business Wire
3 hours ago
- Business Wire
Verantos to Present Practical Approaches in High-Validity Real-World Evidence Generation at ISPE's 41st Annual Meeting
MENLO PARK, Calif.--(BUSINESS WIRE)-- Verantos, the global leader in high-validity real-world evidence at scale, announced today that it will present practical approaches for generating high-validity real-world evidence (RWE) at the International Society for Pharmacoepidemiology (ISPE) 41st Annual Meeting in Washington, D.C. The goal isn't just to measure data reliability; it's to optimize it and to do so using peer-reviewed, published methods so metrics can't be gamed. Share CEO Dan Riskin will share approaches to achieve fit-for-use RWD based on real-world learnings from large pharma collaborations and the FDA-sponsored Verantos-led TRUST and VERIFY studies. He will discuss 'Data reliability: implementing accuracy, completeness, and traceability in practice.' 'The goal isn't just to measure data reliability; it's to optimize it and to do so using peer-reviewed, published methods so metrics can't be gamed,' said Riskin. 'Evidence generated using low-quality or untested RWD remains suspect as it always has been.' Symposium snapshot ISPE 41st Annual Meeting, Washington, D.C. Real-world evidence fit for regulatory applications — what does good look like? Tuesday, August 26, 10:30am EDT Verantos will detail practical implementation approaches to quantify and improve accuracy, completeness, and traceability consistent with peer-reviewed methods and learnings from the TRUST and VERIFY studies. Early findings from this work have been published in JAMA Network Open, providing a transparent foundation for reliability measurement and optimization. About Verantos Verantos is the global leader in high-validity real-world evidence for life sciences organizations. By incorporating robust clinical narrative data and artificial intelligence technology, Verantos is the first company to generate high-validity evidence at scale across therapeutic areas with measured accuracy, completeness, and traceability. Credible evidence accelerates clinical development, market access, medical affairs, pharmacovigilance, and regulatory initiatives.
