Weekly Rundown:  EMA and FDA set shared standards for AI use across drug development

Welcome to the Weekly Rundown where the DDN editors cover this week’s top biotech and pharma news.

EMA and FDA set shared standards for AI use across drug development

European Medicines Agency (EMA) and the FDA have agreed on 10 joint principles to guide the responsible use of AI across the full medicines lifecycle, marking a formal step toward closer transatlantic alignment 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 guidance in both jurisdictions. Regulators said the framework responds to the rapid expansion of AI in drug development by emphasizing risk mitigation, data integrity, and patient safety, while still enabling innovation. In the EU, the principles will feed into forthcoming guidance already in development, building on the EMA’s 2024 AI reflection paper and broader efforts to integrate AI into regulatory decision-making under new pharmaceutical legislation. — Andrea Corona

Killer T cells linked to multiple sclerosis pathology

Nearly one million people in the US are affected by multiple sclerosis (MS), a progressive autoimmune disease where the immune system incorrectly attacks the myelin coating of nerve fibers in the brain and spinal cord. A new study published in Nature Immunology offers insights into how immune cells linked to Epstein-Barr virus (EBV) could contribute to MS pathology. Researchers from the University of California, San Francisco (UCSF) studied the cerebrospinal fluid (CSF) and blood of 13 patients with MS or early signs of the disease, as well as five participants without MS. They found that people with MS had more of certain CD8+ T clonotypes than people without MS. While nearly 95 percent of the population carries EBV, the CD8+ T cells which recognized EBV antigens were between 10 and 100 times more abundant in the CSF than in the blood of people with MS; participants without MS had similar abundance between CSF and blood. Due to the identify-and-destroy nature of CD8+ killer T cells, this could indicate that there is something driving the overactive immune response in the CSF. “The big hope here is that if we can interfere with EBV, we can have a big effect, not just on MS but on other disorders, and improve the quality of life for many, many people,” said Joe Sabatino, neurologist and corresponding author from UCSF, in the press release. – Melissa Kay

Experts warn new vaccine guidance could put children at risk

US childhood vaccination policy faces growing uncertainty as changes to the Advisory Committee on Immunization Practices (ACIP) and Department of Health and Human Services (HHS) recommendations have sparked concern among public health experts. Since mid‑2025, new ACIP appointees with questioned scientific credentials have shifted the focus from universal vaccination toward a reduced schedule, moving vaccines for hepatitis B, COVID-19, influenza, and rotavirus into a “shared clinical decision-making” category. Critics warn this approach, which cites Denmark and other European programs as models, fails to account for fundamental differences in healthcare access, screening, and infant health in the US, where rates of prematurity, chronic illness, and missed screenings remain high. Pediatricians and public health organizations caution that these changes could reduce vaccine uptake, increase preventable infections, and erode public confidence, leaving American children more vulnerable even as the nation’s historical leadership in immunization is called into question. – Bree Foster

Novo Nordisk advances combination therapy with promising trial outcomes

Novo Nordisk’s next-generation weight‑loss drug CagriSema outperformed its blockbuster GLP-1 Wegovy in controlling blood sugar in a late-stage Phase 3 trial, though it still fell short of the company’s ambitious 25 percent weight‑loss goal. In the REIMAGINE 2 study of over 2,700 adults with type 2 diabetes, weekly injections of 2.4mg of CagriSema lowered HbA1c by 1.91 percentage points, compared with a 1.76-point drop for semaglutide and a 0.09-point rise in placebo. Patients also lost an average of 14.2 percent of their body weight over 68 weeks, versus 10.2 percent for semaglutide. While the average weight loss fell below Novo Nordisk’s target, the trial showed that 43 percent of participants achieved at least 15 percent weight loss and 24 percent reached 20 percent or more, highlighting meaningful results for a substantial portion of patients. CagriSema combines the GLP-1 receptor agonist semaglutide with the long-acting amylin analog cagrilintide, and Novo noted the therapy was well-tolerated with no new safety concerns. The company has filed for FDA approval for weight-loss treatment in patients with obesity or who are overweight with comorbidities, and additional trials are ongoing for type 2 diabetes and cardiovascular risk reduction. – Bree Foster

Funding bill curbs PBM practices, locks in telehealth, hospital-at-home extensions

A short-lived government shutdown ended Tuesday with legislation that reinstates Medicare telehealth flexibilities and extends the Acute Hospital Care at Home program, offering rare multi-year certainty for providers after years of temporary waivers. The funding package, which keeps the HHS operating through September 30, preserves expanded Medicare telehealth reimbursement through 2027 and extends hospital-at-home authority through 2030, easing operational and investment pressures for health systems that rely on the programs. Beyond telehealth, the bill delays Medicaid disproportionate share hospital payment cuts until fiscal 2028, advances site-neutral payment policies for off-campus outpatient departments, and enacts new restrictions on pharmacy benefit managers (PBM), including transparency requirements and limits on compensation tied to drug list prices. While pharmacy groups praised the PBM provisions, industry lobbyists argued the reforms will not lower drug costs, underscoring continued disagreement over how Congress should rein in prescription spending even as telehealth policy gains firmer footing.– Andrea Corona

GSK returns rights to Wave’s lead RNA editing program

GSK has handed back global rights to WVE-006, the most advanced RNA editing drug in clinical testing, allowing Wave Life Sciences to take full control of development for the rare disease alpha-1 antitrypsin deficiency. Wave said the decision was not driven by doubts about the drug’s potential but by a strategic alignment that positions the biotech to move faster in rare diseases, while GSK remains focused on larger respiratory indications. The move comes ahead of new clinical data expected by the end of March, with Wave planning regulatory discussions this year around an accelerated approval pathway. WVE-006 is the first RNA editing therapy to reach human trials and has shown early evidence of restoring functional protein production in patients, though investor expectations around potency have remained high amid broader uncertainty in the emerging RNA editing field. – Andrea Corona