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The U.S. Food and Drug Administration proposed on Monday a new framework to speed approvals of personalized treatments for rare and life-threatening genetic diseases, allowing drugmakers to rely on small, well-controlled studies when traditional trials are not possible. The plan aims to update approval standards for individualized genetic medicines, creating a pathway for therapies aimed at patient groups too small for traditional trials. It could provide patients earlier access to treatments that would otherwise stall because only limited data can be gathered, with safety still monitored through ⁠post-approval requirements. The proposal offers drugmakers clearer rules for developing genome-editing and RNA-based drugs, while requiring them to justify why ⁠randomized trials are not feasible, collect real-world evidence after approval and have confirmatory studies underway for accelerated clearance. The agency warned it could withdraw products if those studies fail or are not completed. The proposal offers drugmakers clearer rules for developing genome-editing and RNA-based drugs, while requiring them to justify why ⁠randomized trials are not feasible, collect real-world evidence after approval and have confirmatory studies underway for accelerated clearance. The agency warned it could withdraw products if those studies fail or are not completed. The draft guidance, first outlined in November, would permit companies to seek approval...

AstraZeneca has secured a key regulatory win in its effort to reclaim the lead in the BTK inhibitor market, with the FDA approving its combination of Calquence plus Venclexta as the first all-oral, fixed-duration regimen for first-line chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL). While patients with newly diagnosed CLL typically take a BTK drug like Calquence indefinitely until disease progression, the new approval offers a better quality-of-life alternative by limiting treatment to 14 months. “The continuous regimens frequently used to treat chronic lymphocytic leukemia often come with side effects that may become burdensome to patients over time,” Jennifer Brown, M.D., Ph.D., from the Dana-Farber Cancer Institute, said in a Feb. 20 statement, adding that the Calquence combo gives doctors greater flexibility to tailor treatment plans. The approval arrives as BeOne Medicines continues to widen its lead in the BTK inhibitor space with Brukinsa (zanubrutinib). While AZ hopes its time-limited option will sway clinicians, BeOne leadership has historically dismissed any threat from the Calquence regimen, betting instead on its own BTK/BCL-2 candidate. Brown is the principal investigator of the phase 3 Amplify trial that supported the FDA decision on Calquence. In the study, patients who received Calquence (acalabrutinib) and AbbVie and Roche’s...

The U.S. Food and Drug Administration has approved Vanda Pharmaceuticals' drug for the treatment of two serious mental health conditions, the company said on Friday. The drug, branded as Bysanti, is an antipsychotic pill aimed at treating schizophrenia and acute bipolar I disorder. The approval comes with the FDA's most serious warning of an increased risk of death in elderly patients with dementia-related psychosis. Bysanti, chemically known as milsaperidone, belongs to a class of drugs known as atypical antipsychotics that work by blocking dopamine and serotonin receptors in the brain, which help regulate mood, stress, delusions and alertness. Schizophrenia is a serious mental illness that can cause hallucinations and delusions, while bipolar disorder is marked by dramatic shifts in mood and energy that can disrupt sleep, judgment and daily functioning. Vanda said it expects to launch the drug in the U.S. in the third quarter of this year.

The U.S. Food and Drug Administration said on Thursday it has approved labeling changes to six menopause hormone therapies to remove references to risks of cardiovascular disease, breast cancer, and probable dementia. The FDA had initiated the removal of the strictest "black box" warnings in November in a move to boost access to these treatments long shunned by patients and doctors over safety fears. The agency said 29 drug companies have submitted proposed labeling changes. The first group of six hormone therapies includes estrogen-only and progestogen-only products such as Prometrium, Divigel, Cenestin and Enjuvia, along with the combination therapy Bijuva and the topical vaginal estrogen treatment Estring. Hormone replacement therapy, or HRT, replenishes the hormones, primarily estrogen, that decline with menopause to relieve symptoms like hot flashes and vaginal dryness. HRT was widely prescribed, including to protect women from chronic diseases, especially heart disease, for decades. But its use plunged after a 2002 Women's Health Initiative study found it could raise the risk not only of breast and ovarian cancer, but also of strokes and other serious conditions. Of about 41 million U.S. women aged 45 to 64 in 2020, only about 2 million received a hormone-therapy prescription, the agency said.

The U.S. Food and Drug Administration said a television advertisement for Novo Nordisk's weight-loss pill is "false or misleading", according to a letter dated February 5. The health regulator said the television spot misleadingly suggests that Wegovy in its pill form offers an advancement or improvement over other weight-loss drugs that belong to the ⁠class known as GLP-1s. The FDA also said the claims "live lighter" and "a way forward" misleadingly imply additional weight loss compared to other currently approved GLP-1 treatments, when this has not been demonstrated. The Danish company did not immediately respond to Reuters request for comment. "Additionally, they misleadingly imply benefits beyond physical weight loss, such as emotional relief, reduced psychological burden, hope, or direction for patients' lives, positioning the drug as a solution to broader ⁠life challenges rather than a treatment for a specific condition, when this has also not been demonstrated," the agency said in the letter. The ‍agency said ⁠the Novo ad was in violation of the Federal Food, Drug, and Cosmetic Act, and ⁠asked the drugmaker to take immediate action to address ‌any violations.

The U.S. Food and Drug Administration said on Friday it would take action against telehealth provider Hims & Hers for its $49 weight-loss pill, including restricting access to the drug's ingredients and referring the company to the Department of Justice for potential violations of federal law. Federal action to limit the production of compounded drugs would be a win for Novo Nordisk, which has been losing share in the weight-loss market to rival Eli Lilly and telehealth firms like Hims. However, the FDA did not make clear whether it could quickly halt the sale of Hims' product, the cheapest GLP-1 therapy on the U.S. market. Shares of Hims fell 14.1% in after-hours trade. The FDA said it plans to restrict GLP-1 ingredients used in non-approved compounded drugs that companies such as Hims and other compounding pharmacies have marketed as alternatives to authorized treatments. The agency cited concerns over quality, safety and potential violations of federal law. On Thursday, Reuters was first to report that Hims would begin offering copies of Novo Nordisk's new Wegovy pill at an introductory price of $49 per month, about $100 less than the brand name. Novo Nordisk later said it would take legal action against the telehealth company over...

The U.S. Food and Drug Administration today approved the Zycubo (copper histidinate) injection as the first treatment for Menkes disease in pediatric patients. “With today’s action, children with this devastating, degenerative disease will have an FDA-approved treatment option and the potential to live longer,” 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 FDA will continue to work with the rare disease community to advance drug development for patients with Menkes disease and other rare conditions.” Menkes disease is a neurodegenerative disorder caused by a genetic defect that impairs a child’s ability to absorb copper. The disease is characterized by seizures, failure to gain weight and grow, developmental delays, and intellectual disability. It leads to abnormalities of the vascular system, bladder, bowel, bones, muscles, and nervous system. Children with classical Menkes (90% of those with the disease) begin to develop symptoms in infancy and typically do not live past three years. It affects approximately one in every 100,000-250,000 live births worldwide and is more common in boys.

The U.S. Food and Drug Administration on Wednesday said it is aiming to reduce the number of human clinical studies required for approval of certain biosimilar drugs and cut development costs for the medicines made using living cells. The agency released draft guidance proposing ways to accelerate the availability of less expensive close copies of complex biotech medicines. The move is part of a broader push to cut healthcare costs, U.S. Secretary of Health and Human Services Robert F. Kennedy Jr. said in a press conference. "Under this new framework, companies may not always need to conduct large, expensive human trials when advanced testing can already prove that biosimilars work just as effectively and just as safely as the original drug," Kennedy said. Unlike cheap generic versions of simple-to-manufacture pills, medicines made from living cells cannot be exactly copied so are referred to as biosimilars. Major pharmaceutical companies and industry groups have lobbied against such changes that they argue could hurt innovation and limit treatment options. Biotech drugs are the fastest-growing class of medications in the United States and account for a substantial and growing portion of healthcare costs, the FDA has said. Biosimilars have faced multiple barriers, including physician hesitancy, reimbursement policies, and complex...

The U.S. Food and Drug Administration has flagged three of Philips' (PHG.AS), opens new tab medical device facilities after inspections found they had failed to meet required manufacturing standards, according to an update on the agency's website on Tuesday. The FDA issued a warning letter to the Dutch medical device maker about the facilities at Bothell, Washington and Reedsville, Pennsylvania in the U.S., and Eindhoven in the Netherlands. The devices made at these facilities are considered "adulterated" under U.S. law due to non-compliance with current good manufacturing practices, the FDA said in the letter. U.S.-listed shares of the medical device company were down nearly 5% in morning trading. The inspected sites produce various types of medical equipment, including ultrasound machines, their components, as well as software used for cardiac assessments and patient monitoring. Philips said it takes the warning "very seriously" and has submitted a formal response to the FDA, in line with regulatory requirements. The company continues to manufacture and sell these products, and that it does not expect any material commercial impact from the warning letter, it added. "This seems a relatively minor issue in which Philips did not correctly adhere to the required complaint documentation," said Marc Hesselink, analyst at ING FM. "We...

Germany's Bayer said on Friday the U.S. health regulator has approved its drug for menopause relief. The drug, branded as Lynkuet, is a non-hormonal treatment designed to relieve moderate-to-severe vasomotor symptoms, also known as hot flashes, associated with menopause. The U.S. Food and Drug Administration's approval was based on three late-stage studies that showed Lynkuet reduced the frequency and severity of hot flashes and eased sleep disturbances in menopausal women. The regulator had in July extended the review of the drug, but did not raise any concerns regarding its general approvability.