Fda

• The FDA has approved Lipfendra, a new cholesterol-lowering pill developed by Merck. • Unlike traditional statins, Lipfendra is a PCSK9 inhibitor, utilizing a different mechanism to reduce cholesterol levels in patients. • This approval provides a new oral therapeutic option for managing high cholesterol, offering an alternative for those who may not respond well to statins.

Move comes as prediction platforms rapidly gain popularity and offer opportunities to bet on virtually anythingKalshi is expanding its wagers to include bets on drug trial results, an expansion into new predictions territory as platforms continue to rapidly gain popularity and offer opportunities to bet on virtually anything.The betting platform said the expansion into clinical trials and FDA regulatory decisions would help surface information on drug trials that otherwise go unreported. A publicly listed contract on a drug trial would produce a “continuously updated, public probability that reflects the weight of the evidence, rather than the preferred message of the trial sponsor”, the company said. Continue reading...

• The FDA has introduced a new fast-track program for the approval of biologics, including experimental gene therapies for conditions like sickle cell disease and cancer. • Traditionally, the agency requires a rigorous three-phase trial process involving healthy volunteers, dose-finding studies, and large-scale clinical trials to ensure safety. • Critics argue that these political shortcuts may undermine the FDA's international reputation and its standing as a global authority on drug safety.

• Leading researchers are warning that a recent FDA policy shift may lead to the approval of ineffective medicines. • In February, the FDA announced that drug sponsors could seek approval based on a single clinical trial and confirmatory evidence, departing from a 1960s-era requirement for two clinical studies. • This change is concerning to experts because it may allow drugs with conflicting trial data to enter the market, potentially compromising patient safety and efficacy.

• Public health officials have issued an alert regarding a multistate rise in cyclosporiasis, a parasitic gastrointestinal illness, with over 450 reported cases across 18 states. • Michigan is the most heavily impacted region, accounting for more than 300 of the total cases, though officials warn the actual number is likely higher due to underreporting. • The report also highlights critical medical developments, including safety monitoring for psychedelic therapies by a Yale psychiatrist and new FDA approvals for gene, cell, and nephrology therapies.

• The FDA approved five first-in-class therapies during the first half of 2026, targeting obesity, AML, kidney disease, hearing loss, and bladder cancer. • On April 14, Travere Therapeutics received approval for sparsentan (Filspari), the first treatment specifically approved for focal segmental glomerulosclerosis (FSGS), a rare kidney disease. • These approvals are significant as they replace off-label or burdensome parenteral treatments with more accessible options, such as all-oral regimens that allow patients to be treated at home.

• The FDA has approved the first generic version of baloxavir tablets for the treatment and postexposure prophylaxis of influenza. • The medication is indicated for patients aged 5 years or older, including those who have been exposed to an individual with the flu. • This approval is based on the generic product meeting regulatory standards for equivalence to the reference listed drug rather than new clinical efficacy data.

• The Trump administration has released its 2026 regulatory agenda, which includes several key initiatives focused on the pharmaceutical industry. • Acting FDA Commissioner Kyle Diamantas emphasized a renewed focus on the agency's core mission, specifically regarding medications, diagnostics, treatments, and medical devices. • This agenda signals a strategic shift in how the FDA approaches disease treatment and regulatory oversight for industry stakeholders and patient advocates.

• The FDA issued several decisions in June 2026 regarding new neurology therapeutic agents, including new drug application acceptances and regulatory alignments. • Key Duchenne Muscular Dystrophy (DMD) actions include the acceptance of Sarepta’s sNDAs to transition casimersen and golodirsen to traditional approvals. • A critical Advisory Committee (AdComm) meeting has been scheduled for July 29 to review the Biologics License Application (BLA) for deramiocel.

• The FDA experienced a slight slowdown in regulatory productivity during the first half of 2024 due to significant leadership instability and ongoing workforce attrition. • Under acting commissioner Kyle Diamantas, the agency has shifted toward a more open stance, particularly regarding rare disease therapies and accelerated approval applications. • Key beneficiaries of this shift include REGENXBIO, which was told its clinical data for RGX-121 was sufficient, as well as Replimune and uniQure.

• The FDA approved six novel drugs for pediatric patients under 18 in 2026, including Copper histidinate (Zycubo) by Sentynl Therapeutics Inc. • Approved on January 12, 2026, Zycubo is indicated for the management of Menkes disease in children via subcutaneous formulation. • Clinical data from two open-label trials showed that patients starting treatment within four weeks of birth experienced a 78% reduction in the risk of death.

• Between June 27 and July 4, 2026, the FDA issued two major approvals: expanding Casgevy gene therapy for sickle cell disease patients aged 2 and older, and approving Tregzi, the first Treg cell immunotherapy for chronic graft-versus-host disease. • The roundup also tracks critical healthcare developments across the US, UK, Canada, Australia, and the EU, including NHS funding updates, EMA regulatory decisions, and various biotech deals. • These updates are significant for clinicians, biotech investors, and policy watchers as they signal advancements in gene therapy and hospital consolidation trends.

Experts say the Food and Drug Administration is not prepared for the health threat of bacterial contaminationMultiple brands of infant formula have been recalled recently due to bacterial contamination, and experts say the Food and Drug Administration is inadequately prepared to deal with the health threat they pose in the wake of Trump administration cuts.Last March, the FDA announced the launch of Operation Stork Speed, specifically intended to “expand options for safe, reliable, and nutritious infant formula for American families”. Two months later, Martin Makary, who was FDA commissioner at the time, told Congress that the FDA had lost around 3,100 employees due to the Trump administration’s reorganization and cuts. Makary departed the FDA the same month. Continue reading...

• The FDA updated a Salmonella outbreak warning on June 30, 2026, reporting 119 illnesses across 36 states, including 32 hospitalizations. • The expanded recall now includes products from TNVitamins, Live it Up, Doctor's Pride, and Why Not Natural. • This escalation highlights significant public health risks associated with contaminated moringa supplements.

• On June 2, 2026, the FDA approved ensitrelvir for the post-exposure prophylaxis of COVID-19 in patients aged 12 years and older. • The regulatory decision follows positive results from phase 3 clinical trials, confirming the drug's efficacy in preventing infection. • This approval provides clinicians with a critical new tool to limit the spread of the virus among adolescents following close-contact or household exposure.

An estimated 650,000 bags of potato chips are affected as US agency upgraded recall of several popular brandsThe US Food and Drug Administration (FDA) has upgraded a recall of several popular brands of potato chips to its most serious level because of the risk of salmonella contamination.Manufacturer Utz issued a voluntary recall in May for varieties of its Zapp’s and Dirty potato chips products, citing the possible presence of salmonella in dry milk powder sourced from a third party used to make a seasoning ingredient. Continue reading...

• The US Food and Drug Administration (FDA) approved 22 novel drugs during the first half of 2026, expanding treatment options across various medical specialties. • Key approvals include copper histidinate (formerly CUTX-101) for Menkes disease, supported by two open-label studies involving 129 patients treated with subcutaneous administration for up to 3 years. • The FDA also granted approval for a new treatment targeting mild to moderate Alzheimer's disease (AD) based on phase 3 clinical trial data from both adult and pediatric populations.

• A recent New England Journal of Medicine (NEJM) pragmatic trial tested a 6-month rifampicin-resistant tuberculosis (RR-TB) regimen, establishing a new benchmark for adaptive evidence generation. • The trial embedded randomization directly into existing treatment infrastructure to bypass the lengthy 18-month site qualification and rigid eligibility criteria typical of conventional Phase 3 development. • This approach addresses a critical public health crisis, as the WHO Global Tuberculosis Report 2025 estimates 390,000 new MDR/RR-TB cases annually and 150,000 drug-resistant TB deaths in 2024.

• The FDA approved the updated COVID-19 vaccine strain in May, but official guidance on who should receive it remains unavailable. • A critical lack of a quorum since March has stalled the committee responsible for determining eligibility and insurance coverage for the fall rollout. • This administrative gap creates uncertainty for healthcare providers and patients regarding the timing and necessity of booster shots.

• Paradigm Health formally submitted its response to the U.S. Food and Drug Administration's (FDA) Request for Information (RFI) regarding the Real-Time Clinical Trial (RTCT) Initiative on June 30, 2026. • As the sole technology provider currently operationalizing the FDA's RTCT proof-of-concept studies, Paradigm Health based its submission on insights from biopharma sponsors and healthcare providers. • This move is critical for refining the framework of real-time clinical trials, aiming to accelerate drug development and improve the efficiency of regulatory oversight.

• The FDA has placed psilocybin and an MDMA-like drug on an accelerated 1-to-2 month approval track, potentially making psilocybin the first FDA-approved psychedelic therapy. • This fast-track process follows a Trump executive order and the use of priority review vouchers, with full approval possible by late 2026. • The shift is supported by evolving psychiatric science, highlighted by the Chicago VA Hospital recently enrolling its first veterans in a psilocybin-assisted therapy program for PTSD.

• On June 22, the FDA announced several initiatives to accelerate both early- and late-stage drug development as part of the Department of Health and Human Services' "Operation TrailBlazer." • A key component is a new drug pilot program designed to shorten the timeline between initial drug identification and first-in-human clinical studies. • The program aims to establish a network of qualified research institutions, including academic medical centers and contract research organizations, to collaborate with sponsors on protocol development.

• On June 2, 2026, the FDA approved ensitrelvir for the post-exposure prophylaxis of COVID-19 in patients aged 12 years and older. • The regulatory decision follows positive results from phase 3 clinical trials, confirming the drug's efficacy in this demographic. • This approval provides clinicians with a critical new tool to limit the spread of the virus among adolescents following close-contact or household exposure.

Committee will meet in July to discuss peptides now sold in gray market despite limited evidence of safety and efficacyAdvisers to the Food and Drug Administration (FDA) will soon hold a meeting about whether to ease restrictions on access to some research peptides, a group of drugs with a zealous following and thin evidence to support them.If restrictions are eased, US compounding pharmacies would be able to produce and fill prescriptions for Americans – a change that would effectively legalize a thriving gray market. Continue reading...

• White House deputy assistant for domestic policy Heidi Overton is reportedly among the final candidates being considered to lead the Food and Drug Administration (FDA). • Overton is a medical doctor who holds a doctorate in clinical investigation from Johns Hopkins University, bringing significant medical and policy expertise to the role. • This potential appointment is critical as the FDA head oversees the safety and efficacy of pharmaceuticals and food products across the United States.

• The FDA has launched a new initiative aimed at resetting the drug review process to accelerate the development of new medications. • A primary goal of the program is to boost the number of domestic clinical trials, reducing reliance on overseas data and speeding up patient access to therapies. • This shift matters as the agency seeks to streamline regulatory hurdles and improve the efficiency of bringing innovative treatments to the U.S. market.

• The US Administration is restructuring the systems supporting PEPFAR and global health security, shifting its engagement strategy toward regulatory influence over long-term public health partnerships. • Concerns are rising that increased political oversight and the deconstruction of international collaborations may undermine the NIH's scientific infrastructure and its history of HIV research and vaccine development. • In a move toward regulatory cooperation, the US Food and Drug Administration (FDA) and the African Medicines Agency (AMA) signed a Memorandum of Understanding (MoU) to establish a shared framework for information sharing.

• The FDA has accepted Roche's application to use the combination of Lunsumio and Polivy for patient treatment. • This regulatory milestone is part of Roche's effort to expand the indications and clinical utility of these existing in-market products. • The development matters as it potentially provides a new therapeutic option for patients requiring this specific drug combination.