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The U.S. Food and Drug Administration has approved Precigen's (PGEN.O), opens new tab immunotherapy to treat adults with a rare respiratory disease, making it the first treatment for the condition to receive the health regulator's nod. Shares of the company surged more than 81% to $3.36 in premarket trading on Friday. Keep up with the latest medical breakthroughs and healthcare trends with the Reuters Health Rounds newsletter. Sign up here. The therapy, Papzimeos, was approved to treat recurrent respiratory papillomatosis (RRP) — a condition that causes growth of wart-like tumors in the respiratory tract due to human papillomavirus (HPV) infection. RRP can be fatal as there is no cure and the current standard-of-care is repeated surgeries. A distinguishing aspect of this disease is the tendency for the growth to return even after removing them through surgical procedures. "Everybody is anxiously awaiting a new treatment for this disease. The patients are and the surgeons are. There's nothing more frustrating than doing a surgery and then having the patient come back six months later," said Simon Best, associate professor of Otolaryngology at Johns Hopkins Hospital. Precigen estimates about 27,000 adult RRP patients in the U.S. It did not immediately respond to a Reuters request for comment on...

The U.S. Food and Drug Administration has approved Tonix Pharmaceuticals' (TNXP.O), opens new tab drug to manage pain related to a type of chronic condition called fibromyalgia, the company said on Friday. The approval paves the way for the drug to be the first new treatment option in over 15 years for patients with fibromyalgia, which causes widespread pain, fatigue as well as problems with sleep and memory. Keep up with the latest medical breakthroughs and healthcare trends with the Reuters Health Rounds newsletter. Sign up here. Patients have so far relied on older drugs such as Lyrica, Cymbalta and AbbVie's (ABBV.N), opens new tab Savella. The non-opioid drug, which will be sold under the brand name Tonmya, is a pill designed for bedtime treatment to improve sleep quality and reduce pain associated with the chronic condition. "This is the first time that a drug has been developed to target the non-restorative sleep, which we think plays an important role in fibromyalgia," CEO Seth Lederman said in an interview with Reuters ahead of the decision. Tonix's application to the health regulator was based on two late-stage studies, in which the drug significantly reduced daily pain compared to placebo at 14 weeks. Fibromyalgia has long been misunderstood...

The FDA granted fast track designation to NRX-100 (NRx Pharmaceuticals, Inc) a ketamine-based drug, as a standalone therapy for the treatment of suicidal ideation in individuals with depression, including bipolar depression. "We thank FDA for its thoughtful review of our fast track designation request and believe this regulatory determination is a significant step forward in our goal to address the national crisis of suicide among soldiers, first responders, veterans, and civilians alike." Jonathan Javitt, MD, MPH, chairman and CEO of NRx Pharmaceuticals, said in a news release. NRX-100 in Suicidal Ideation According to the CDC, nearly 13 million individuals seriously consider suicide annually, with around 1.5 million attempts, marking suicide as the leading cause of death that impacts individuals of all ages. Suicide rates in the United States increased by approximately 36% between 2000 and 2022. The number of individuals impacted by suicidal thoughts and actions is much higher, with an estimated 12.8 million adults seriously considering suicide, 3.7 million planning, and 1.5 million attempting suicide in 2022 alone. In 2023, there were 49,316 deaths from suicide, which amounts to roughly 1 death every 11 minutes. Further data from 2023 identified that suicide was among the top 8 leading causes of death...

Using a series of machine learning models and laboratory experiments, investigators found a series of FDA-approved drugs that could provide lipid-lowering properties to patients dealing with high cholesterol, according to research published in Acta Pharmacologica Sinica. If successfully repurposed, these drugs could expand the arsenal of lipid-lowering therapies available for clinicians and pharmacists to provide to patients. Critical Need for Novel Agents to Treat Hyperlipidemia Hyperlipidemia carries a high presence in the US population. The American Heart Association’s 2023 Heart Disease and Stroke Statistics Update found a 34.7% prevalence of hypercholesterolemia and about a quarter of US adults harbored elevated low-density lipoprotein cholesterol (LDL-C) levels from 2017 to 2022. The role of elevated LDL-C in the development of cardiovascular disease necessitates that thorough vigilance and comprehensive management of hyperlipidemia be employed for patients. A variety of lipid-lowering therapies are available to patients in the form of statins, cholesterol absorption inhibitors, and proprotein convertase subtilisin/kexin type 9 (PCSK9) inhibitors. These medications, each with their own unique mechanism of action, are effective at reducing lipid levels, but patients face a multitude of challenges regarding uptake of such medications. Studies have found that some individuals demonstrate poor tolerance to existing therapies, whereas others exhibit reduced...

British drugmaker GSK said on Monday that the U.S. Food and Drug Administration has accepted its application for priority review of gepotidacin, an oral antibiotic to treat sexually transmitted uncomplicated gonorrhoea. GSK is counting on new infectious disease treatments, such as its recently launched respiratory syncytial virus vaccine, to help offset revenue losses from its top-selling drugs and anticipated patent expirations for its HIV therapies. The drug gepotidacin has already been approved in the United States under the brand name Blujepa for a common type of urinary tract infection (UTI) in women and adolescent girls. The FDA's decision on the drug's treatment of uncomplicated urogenital gonorrhoea is expected in December. If approved, it would provide an oral option to patients who currently rely on injectable treatments.

The Food and Drug Administration (FDA) has approved Skytrofa® (lonapegsomatropin-tcgd) for the replacement of endogenous growth hormone in adults with growth hormone deficiency (GHD). Lonapegsomatropin is a pegylated prodrug of the human growth hormone, somatropin, administered as a subcutaneous injection once weekly. It is designed to provide a sustained release of active, unmodified somatropin. The product was previously approved for the treatment of pediatric patients 1 year and older who weigh at least 11.5kg and have growth failure due to inadequate secretion of endogenous growth hormone. The approval in adult GHD was based on data from the randomized, parallel-arm, placebo-controlled and active controlled foresiGHt trial (ClinicalTrials.gov Identifier: NCT04615273), which included 259 patients aged 23 to 81 years. Study participants were randomly assigned 1:1:1 to receive lonapegsomatropin once weekly (n=89), placebo once weekly (n=84), or somatropin once daily (n=86). The primary endpoint was the change in trunk percent fat from baseline to week 38 as assessed by dual X-ray absorptiometry.

A shortage of intravenous and injectable saline solutions in the United States has now been resolved, U.S. Food and Drug Administration commissioner Martin Makary said on Friday. A shortage of IV fluids, which provide nutrients to the body or replenish fluids lost due to injury or illness, had forced hospitals to curtail elective procedures last year. The shortage partly stems from Baxter International's (BAX.N), opens new tab North Carolina plant getting damaged in Hurricane Helene in September. At that time, the Marion, North Carolina site was producing 60% of the nation's supply of IV fluids and peritoneal dialysis solutions, amounting to 1.5 million bags daily, according to the American Hospital Association. Baxter and the FDA did not immediately respond to Reuters' requests for comment. Germany's Fresenius (FMEG.DE), opens new tab and B. Braun Medical had ramped up their production to help mitigate these shortages, while Baxter worked with the FDA to import some intravenous products. Last week, the company said demand remained subdued, even as supply stabilized. In May, Baxter said inventory levels at its North Carolina facility had been fully restored. The FDA said it is working closely with manufacturers and will continue to monitor the supply of other IV fluids, which are still in...

The FDA has approved a label update for inclisiran (Leqvio; Novartis Pharmaceuticals), allowing its use as monotherapy, in addition to diet and exercise, to reduce low-density lipoprotein cholesterol (LDL-C) in adults with hypercholesterolemia. This first-line label update reinforces proven ability to effectively lower LDL-C, a critical risk factor for heart disease," Victor Bultó, president of US Novartis, said in a news release. Inclisiran’s Indications and Approval Inclisiran was originally approved by the FDA in December 2021 as an injection with diet and maximally tolerated statin therapy for adults with heterozygous familial hypercholesterolemia (HeFH) or clinical atherosclerotic cardiovascular disease (ASCVD). It is indicated as a 284-mg dose administered under the skin, with a second dose at 3 months and continued treatment once every 6 months. The effectiveness of inclisiran was studied in 3 randomized, double-blind, placebo-controlled trials that included a total of 3457 individuals with HeFH or clinical ASCVD. The primary measure of effectiveness across all studies was the percentage change in LDL-C from the start of the trial to day 510. Across all 3 studies, individuals received subcutaneous injections of either 284 mg of inclisiran or a placebo on days 1, 90, 270, and 450.2. A total of 1561 adults with ASCVD were...

The U.S. Food and Drug Administration on Thursday announced a new program to speed up construction and review of drug manufacturing plants in the country to boost domestic drug supply. The program, called FDA PreCheck, aims to streamline review of domestic pharmaceutical plants and eliminate unnecessary regulatory requirements, in line with President Donald Trump's executive order in May to shift manufacturing of drugs to the United States. Keep up with the latest medical breakthroughs and healthcare trends with the Reuters Health Rounds newsletter. Sign up here. "The FDA PreCheck initiative is one of many steps FDA is taking that can help reverse America's reliance on foreign drug manufacturing and ensure that Americans have a resilient, strong, and domestic drug supply," FDA Commissioner Marty Makary said in a statement. The regulator in June launched a program to incentivize drug developers that align with national priorities, including increased domestic manufacturing, with considerably shorter review times for their marketing applications. Several major drugmakers, including AstraZeneca (AZN.L), opens new tab, Eli Lilly (LLY.N), opens new tab and Johnson & Johnson (JNJ.N), opens new tab, have pledged billions of dollars to scale up their U.S. footprint. The Trump administration has threatened to impose tariffs on pharmaceutical imports, starting "small" and...

The U.S. Food and Drug Administration said on Friday it has approved Boehringer Ingelheim's drug for patients with a type of advanced lung cancer who have received prior treatment. The drug, branded as Hernexeos, is intended for previously treated patients with non-squamous non-small cell lung cancer whose tumors have a specific genetic mutation. The regulator's approval was based on a study in which Hernexeos helped about 75% of patients who received prior chemotherapy achieve a complete disappearance of cancer or reduction in tumor size. The FDA also approved Life Technologies' diagnostic device to identify patients eligible for the treatment. Hernexeos belongs to a class of drugs known as kinase inhibitors, which target mutations in specific proteins in the body that lead to abnormal cell growth. The drug comes with warnings for possible liver damage, heart problems, lung inflammation and risks to unborn children. The recommended dose depends on the patient's weight and is taken once daily.