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The U.S. Food and Drug Administration on Wednesday proposed a new process to streamline the approval of drugs targeting rare diseases with very small patient populations, including cases supported by data from single-arm clinical trials. The regulator said eligibility will be based on whether the disease affects a small population – fewer than 1,000 individuals in the United States – and is intended to treat a genetic defect. It will also depend on whether there are no adequate alternative therapies that alter the course of the disease. The agency added that it expects substantial evidence of effectiveness may generally be established based on one adequate study, which may be a single-arm trial. This will be supported by data that provides confirmatory evidence of the drug's treatment effect, which could be data on its action at the target site and pre-clinical data. FDA's proposal comes after Health Secretary Robert F. Kennedy Jr. said in June that the U.S. drugs regulator would look for ways to fast-track approval for rare disease treatments and remove obstacles to their path to market. The appointment of Vinay Prasad, the FDA's top vaccine and biologics official, as the head of the FDA's Center for Biologics Evaluation and Research had stoked fears...

Drug developers are increasing adoption of AI technologies for discovery and safety testing to get faster and cheaper results, in line with an FDA push to reduce animal testing in the near future. Within the next three to five years, using AI and cutting back on animal testing could reduce timelines and costs by at least half, according to 11 different experts from across contract research firms, biotech companies and brokerages. Drug development software maker Certara, and biotechs such as Schrodinger and Recursion Pharmaceuticals are already using AI to predict how experimental drugs might be absorbed, distributed, or trigger toxic side effects. "We are getting to the point where we don't actually need to do that (animal testing) anymore," said Patrick Smith, president of drug development solutions at Certara, which works with companies developing infectious diseases drugs such as monoclonal antibodies for hepatitis B. Recursion said its AI-based drug discovery platform took just 18 months to move a molecule into clinical testing as a cancer drug candidate, far faster than the industry average of 42 months. Analysts at TD Cowen and Jefferies expect these AI-driven approaches to cut costs and timelines by more than half, from current estimates of up to 15 years and...

The U.S. Food and Drug Administration on Wednesday proposed a new process to streamline the approval of drugs targeting rare diseases with very small patient populations, including cases supported by data from single-arm clinical trials. The regulator said eligibility will be based on whether the disease affects a small population – fewer than 1,000 individuals in the United States – and is intended to treat a genetic defect. Keep up with the latest medical breakthroughs and healthcare trends with the Reuters Health Rounds newsletter. Sign up here. It will also depend on whether there are no adequate alternative therapies that alter the course of the disease. The agency added that it expects substantial evidence of effectiveness may generally be established based on one adequate study, which may be a single-arm trial. This will be supported by data that provides confirmatory evidence of the drug's treatment effect, which could be data on its action at the target site and pre-clinical data. FDA's proposal comes after Health Secretary Robert F. Kennedy Jr. said in June that the U.S. drugs regulator would look for ways to fast-track approval for rare disease treatments and remove obstacles to their path to market. The appointment of Vinay Prasad, the FDA's top vaccine...

The FDA issued a public alert on Tuesday concerning generic versions of a widely-used hair loss drug that is frequently distributed by popular telehealth brands like Hims and Keeps. The agency cited 32 reports of adverse events that involved compounded topical finasteride products, which are “potentially putting consumers at risk.” These events include “erectile dysfunction, anxiety, suicidal ideation, brain fog, depression, fatigue, insomnia, decreased libido and testicular pain.” The alert specifically targets a spray-on formulation of finasteride, the active ingredient also found in the oral drug Propecia. According to the FDA, these topical versions have not received official approval, and no comprehensive safety data has been submitted for them. At this time, the only FDA-approved oral finasteride products are Proscar and Propecia. The warning follows a Wall Street Journal report in March about men who experienced severe side effects after acquiring finasteride through a telehealth company. One of the men, US Army Sgt. Mark Millich, 26, began dealing with anxiety, dizziness and slurred speech — and then things got even worse. His sex drive plummeted, and his genitals shrank and changed shape. His doctor said it was due to the medication. None of the 17 men the WSJ spoke to who had severe side effects from getting the...

The Saudi Food and Drug Authority (SFDA) has approved an innovative biotechnology-based in vitro diagnostic test designed to aid in early detection of Alzheimer's disease by measuring a specific biomarker in blood plasma. This test represents a groundbreaking advancement in medical laboratory diagnostics, significantly enhancing the early detection of neurodegenerative diseases. Introducing a Faster and More Accurate Diagnostic Solution This diagnostic test measures the concentration of the pTau181 protein in a patient’s blood plasma, providing accurate results in under 20 minutes. This innovative technology marks a significant improvement over traditional diagnostic methods for Alzheimer's disease, which often involve invasive and time-consuming procedures such as cerebrospinal fluid (CSF) analysis or expensive positron emission tomography (PET) scans. A Comprehensive Scientific Evaluation Ensuring Regulatory Compliance The SFDA approved a thorough scientific evaluation conducted by a team of experts. This review included a detailed assessment of the device’s technical and clinical documentation, as well as clinical study data, to confirm its safety, efficacy, and reliability in compliance with regulatory requirements and globally recognized standards. Aligning with Saudi Arabia’s National Healthcare Vision The introduction of this biotechnology-based diagnostic solution strategically enhances the local market by equipping the healthcare sector with accurate and reliable tools for early diagnosis. This advancement will...

The U.S. Food and Drug Administration is now providing real-time, daily publication updates on adverse event data from its reporting system. In an effort to modernize and provide transparency on public health, the agency announced at the end of last week that adverse event data from the FDA Adverse Event Reporting System (FAERS) will be published daily. Previously, updates were published on a quarterly basis. "People who navigate the government's clunky adverse event reporting websites should not have to wait months for that information to become public," FDA Commissioner Marty Makary, M.D., M.P.H., said in a statement. "We're closing that waiting period and will continue to streamline the process from start to finish." The FAERS contains reports from health care professionals, consumers, and manufacturers on adverse events, serious medication errors, and product quality complaints for prescription drugs and biologics. The public can access the data on the FAERS Public Dashboard. According to the FDA, the agency receives more than 2 million reports on adverse events and medication error reports each year. The shift to daily publication is part of "the FDA's broader data modernization strategy to streamline all of its adverse event reporting systems and increase reporting frequency across all systems to identify...

The U.S. Food and Drug Administration has approved Sanofi's (SASY.PA), opens new tab drug to treat a type of blood disorder, the company said on Friday, in a sign that the French drugmaker's $3.7 billion bet on the treatment could start to pay off. The approval aids the company's broader push to become a power house of rare disease and immunology drugs, as it looks to drive growth beyond its top-selling eczema treatment, Dupixent, on which it partners with Regeneron Pharmaceuticals (REGN.O), opens new tab. Sanofi received access to the oral drug, rilzabrutinib, through its deal for Principia Biopharma in 2020. Leerink analysts expect the treatment to bring in peak sales of $2 billion to $5 billion. Rilzabrutinib, to be sold under the brand name Wayrilz, received approval for use in adult patients with the condition known as persistent or chronic immune thrombocytopenia (ITP) who have stopped responding to prior treatments. Sanofi plans to make the drug available to U.S. patients in September. The drugmaker has set a list price of $17,500 for a month's supply of the treatment, but actual costs for patients will vary based on their insurance plans. The condition is a rare blood disorder in which the immune system mistakenly...

The U.S. Food and Drug Administration has approved Teva Pharmaceuticals’ cheaper generic version of Novo Nordisk’s older weight-loss drug, Saxenda, the Israel-based company said on Thursday. Saxenda, chemically known as liraglutide, belongs to the first generation of GLP-1 drugs, which curb appetite as well as help control blood sugar. This results in less weight loss on average than Novo’s popular newer treatment, Wegovy. The FDA approval makes Teva’s version the first generic GLP-1 drug indicated for weight loss in the U.S., the company said. Saxenda is approved for obese adults with weight-related medical problems and pediatric patients aged 12 to 17 years who weigh more than 60 kg and have obesity.

As a result of years of collaborative efforts with King Faisal Specialist Hospital and Research Centre (KFSHRC), the Saudi Food and Drug Authority (SFDA) has approved the registration of the first-of-its-kind clinical study (Phase I) of an investigational gene therapy, developed in Saudi Arabia, using Chimeric Antigen Receptor T-cells (CAR T-cells), under the title: “Phase I Study of Chimeric Antigen Receptor (CAR) T-cells in Adult Patients with Relapsed/Refractory CD19 Positive Acute Lymphoblastic Leukemia (ALL), Using a Closed Transduction System.” Acute Lymphoblastic Leukemia (ALL) is a rapidly progressing cancer of the blood and bone marrow that affects lymphoid white blood cells. It causes the bone marrow to produce large number of immature lymphoblasts, which crowd out normal cells and impair the body’s ability to fight infections, transport oxygen, and control bleeding. The investigational treatment is being developed by a team of scientists and researchers at KFSHRC, in collaboration with Lentigen/Miltenyi Company, and manufactured at the hospital within an internal unit that uses a closed transduction system. The treatment is administered to patients through intravenous infusion. The study's primary aim is to confirm the product's safety in adult patients aged 18 to 60 years. The SFDA affirms that this approval is part of its...

 The U.S. Food and Drug Administration has approved updated COVID-19 vaccines for all people aged 65 and older and for people under 65 with higher health risks, the U.S. health secretary said on Wednesday. The U.S. Department of Health and Human Services did not immediately respond to a Reuters request for comment. The three approved shots are made by Pfizer (PFE.N), opens new tab with German partner BioNTech (22UAy.DE), opens new tab, Moderna (MRNA.O), opens new tab, and Novavax (NVAX.O), opens new tab. Pfizer said the emergency use authorization for its COVID vaccine for children under age 5 had been rescinded. Moderna's shot was approved for ages 6 months and up, Health Secretary Robert F. Kennedy Jr. said. Moderna was not immediately available for comment. Pfizer and BioNTech's updated version of Comirnaty was approved for everyone 65 and over, as well as in individuals ages 5 through 64 years with at least one underlying condition that puts them at high risk for severe outcomes from COVID infection, the company said in a press release.