These will set expectations for how AI should be developed and managed throughout the drug lifecycle and will inform future regulatory guidelines in both jurisdictions.
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Vaccination guidance, promising trial results and funding legislation led the news this week.
Welcome to the Weekly Rundown. DDN Our editors cover the week’s top stories in biotech and pharma.
EMA and FDA set common standards for the use of AI throughout drug development
The European Medicines Agency (EMA) and FDA have agreed on 10 joint principles to guide the responsible use of AI across the drug lifecycle, taking a formal step towards closer transatlantic collaboration on AI regulation. The principles outline expectations for how AI should be developed, validated and managed in activities ranging from early research and clinical trials to manufacturing and post-market safety monitoring, and are intended to inform future regulatory guidelines in both jurisdictions. Regulators said the framework enables innovation while addressing the rapid expansion of AI in drug development by emphasizing risk mitigation, data integrity and patient safety. In the EU, this principle will be reflected in the EMA’s 2024 AI Reflection Document and future guidance already in development, building on broader efforts to integrate AI into regulatory decision-making under the new Pharmaceutical Affairs Act. — andrea corona
Killer T cells associated with multiple sclerosis pathology
Multiple sclerosis (MS) affects nearly 1 million people in the United States. Multiple sclerosis is a progressive autoimmune disease in which the immune system mistakenly attacks the myelin covering of nerve fibers in the brain and spinal cord. New research published in innate immunology We provide insight into how Epstein-Barr virus (EBV)-associated immune cells contribute to MS pathology. Researchers at the University of California, San Francisco (UCSF) studied the cerebrospinal fluid (CSF) and blood of 13 patients with MS or early signs of MS and five participants without MS. They found that MS patients had more of a particular CD8+ T clonotype than people without MS. Almost 95 percent of the population is infected with EBV, but CD8+ T cells that recognize EBV antigens were 10 to 100 times more abundant in the CSF than in the blood of MS patients. Participants without MS had similar CSF and blood abundance. Because CD8+ killer T cells have the ability to identify and destroy, this may indicate that there is something in the CSF that is causing an excessive immune response. “The great hope here is that if we can stop EBV, we can have a profound effect not just on MS, but on other diseases, and improve the quality of life for so many people,” UCSF neurologist and corresponding author Joe Sabatino said in a press release. – melissa kay
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Experts warn new vaccine guidance could put children at risk
U.S. childhood vaccination policy faces increased uncertainty as changes in recommendations from the Advisory Committee on Immunization Practices (ACIP) and the Department of Health and Human Services (HHS) are raising concerns among public health experts. Starting in mid-2025, new ACIP appointees with questionable scientific credentials shifted their focus from universal vaccination to a compressed schedule, moving hepatitis B, COVID-19, influenza, and rotavirus vaccines to the “shared clinical decision-making” category. Critics warn that the approach, which cites Danish and other European programs as models, fails to account for fundamental differences in health care access, screening, and infant health in the United States, where rates of prematurity, chronic disease, and missed screenings remain high. Pediatricians and public health organizations have warned that these changes could reduce vaccine uptake, increase preventable infections, erode public trust and leave America’s children more vulnerable even as the nation’s historic leadership in immunizations is called into question. – Bree Foster
Novo Nordisk advances combination therapy with promising trial results
Novo Nordisk’s next-generation weight loss drug Kaglisema outperformed the company’s blockbuster GLP-1 Wegoby in controlling blood sugar in a late-stage Phase 3 trial, but it still fell short of the company’s ambitious 25% weight loss goal. In the REIMAGINE 2 study of more than 2,700 adults with type 2 diabetes, weekly injections of 2.4 mg of CagriSema lowered HbA1c by 1.91 percent, compared with a 1.76 point decrease with semaglutide and a 0.09 point increase with placebo. Patients also lost an average of 14.2 percent over 68 weeks compared to 10.2 percent with semaglutide. Although average weight loss was below Novo Nordisk’s goals, the trial showed that 43% of participants achieved at least 15% weight loss and 24% achieved weight loss of 20% or more, highlighting meaningful results for a significant proportion of patients. CagriSema combines semaglutide, a GLP-1 receptor agonist, with cagrilintide, a long-acting amylin analog, and Novo said the treatment is well-tolerated and presents no new safety concerns. The company has applied for FDA approval for weight loss treatment in obese or overweight patients with comorbidities, and additional trials are underway in type 2 diabetes and cardiovascular risk reduction. – Bree Foster
Funding bill curbs PBM practices, locks in telemedicine, and curbs home hospital extensions
The brief government shutdown ended Tuesday with legislation restoring Medicare’s telemedicine flexibility and extending the Home Acute Hospital Care Program, providing rare multiyear certainty for health care providers after years of temporary exemptions. The funding package will keep HHS operational through September 30, maintain Medicare telehealth reimbursement expansion through 2027, extend home hospital authority through 2030, and ease operating and investment pressures on health systems that rely on the program. In addition to telemedicine, the bill would also postpone the Medicaid disproportionate hospital share reduction until fiscal year 2028, promote site-neutral payment policies for off-campus outpatient departments, and establish new restrictions for pharmacy benefit managers (PBMs), including transparency requirements and compensation limits related to drug list prices. While pharmacy groups praised the PBM provisions, industry lobbyists argued that the reforms would not lower drug costs, highlighting continued disagreements over how Congress should rein in prescription spending even as telemedicine policy is on stronger footing. andrea corona
GSK returns rights to Wave’s lead RNA editing program
GSK has returned worldwide rights to WVE-006, a cutting-edge RNA editing drug in clinical trials, giving Wave Life Sciences full control over its development for the rare disease alpha-1 antitrypsin deficiency. Weib said the decision was not due to doubts about the drug’s potential, but rather a strategic adjustment to advance biotechnology faster in rare diseases while GSK continues to focus on the larger respiratory disease indications. The move comes ahead of new clinical data expected by the end of March, and Wave plans regulatory discussions this year to accelerate approval. WVE-006 is the first RNA editing therapy to reach human trials and shows early evidence of restoring functional protein production in patients, but investor expectations regarding efficacy remain high amid widespread uncertainty in the emerging RNA editing field. – andrea corona
