Curated news and analysis on clinical trials, drug approvals, and medical research.
The FDA has approved Agios Pharmaceuticals' mitapivat (AQVESME) as the first oral treatment for anemia in thalassemia, reducing transfusion needs by 40-50%. Meanwhile, a clinical trial found a three-times-weekly dosing of vadadustat (Vafseo) is safe for kidney dialysis patients, potentially improving convenience.
A randomized trial found that remote telehealth genetic services increase uptake of genetic counseling and testing in adult survivors of childhood cancer. At six months, 43% of the telehealth group received services vs. 15% in usual care, with actionable results in 10% of those tested.
Semaglutide improves kidney, survival, and quality-of-life outcomes in type 2 diabetes with CKD, per FLOW trial analyses. Benefits were consistent across cardiovascular and severity subgroups, adding eight full-health days per year.
Prostate cancer updates: Erleada combination improves surgical outcomes, Novartis' actinium drug shows PSA responses, and Bayer-Kairos partner to combat Xofigo resistance.
New reports project AI in drug discovery at $17.56B by 2031 (28.1% CAGR) or $2.29B by 2031 (12.49% CAGR). North America led in 2025, oncology topped therapy areas, and talent shortages persist.
The FDA proposes to remove three GLP-1 drugs from the bulk compounding list as adverse events near 2,000 and Senator Tom Cotton demands a probe into Chinese APIs in compounded meds. Shortages have largely resolved, but safety and innovation concerns persist.
FDA granted traditional approval to Novartis' Fabhalta (iptacopan) for IgA nephropathy. Phase 3 APPLAUSE-IgAN data showed eGFR decline slowed by 48% versus placebo over two years.
The FDA has agreed to review Moderna's mRNA flu vaccine after initially rejecting it, with a decision expected by August 2026. The reversal occurs amid a contraction in the vaccine sector, with funding drops and layoffs under the Trump administration.
Two new surveys reveal that 24% of U.S. workers stay in unwanted jobs for health insurance, while 19% of adults with tinnitus have cut hours or quit. Debt and chronic illness increase job lock risk; cognitive behavioral therapy shows promise for tinnitus-related work impact.
Recent Rethinking Clinical Trials Grand Rounds sessions covered the COMPARE-Pediatric IBD study, a hypertension self-management texting intervention for Black Veterans, and the upcoming Pediatric Trials Network presentation.
AstraZeneca to present 85+ oncology abstracts at ASCO 2026. Sun Pharma's Fibromun misses endpoints; Nidlegy hits 52.6% response. Soft tissue sarcoma advances and radiopharma market grows.
A large clinical trial found that adding apalutamide to hormone therapy before and after prostate cancer surgery improves outcomes for high-risk patients. NHS England will roll out SABR radiotherapy requiring only five sessions, and new ctDNA and robotic biopsy technologies are advancing prostate cancer care.
Italian police seized pure cocaine valued at $286 million hidden in a banana shipment from Ecuador at the port of Vado Ligure. The 1,700-pound bust follows a similar seizure earlier this month and underscores the persistent use of banana shipments for international cocaine trafficking.
Gene therapy ended 2025 with renewed funding despite safety setbacks and regulatory cuts. Approvals like Otarmeni and rounds at Kriya, Addition, SonoThera marked momentum; financing and access hurdles persist.
The acute pain market reached $4.5B in 2025, with 98% of pain drugs generic. Voltage-gated sodium channel inhibitors like Journavx offer non-opioid relief, while 39% of the pipeline uses non-small-molecule modalities.
A federal judge blocked grant terminations based on changed priorities; OMB proposes political control over awards. Ph.D. enrollment declines as funding uncertainty persists, and science leaders urge advocacy.
Federated learning models have been shown to outperform all alternatives in drug discovery, while the broader life sciences sector increasingly relies on high-performance computing and AI-ready data infrastructure. These trends are reshaping R&D, with new collaborative approaches and data center demands driving innovation.
The FDA has approved Tregzi, a donor-derived T-cell immunotherapy, to reduce the risk of chronic graft-versus-host disease following stem cell transplant in adults with high-risk blood cancers. In a pivotal trial, 78% of recipients were alive and free of severe chronic GVHD at one year versus 38.4% with standard transplant.
Helus completed Phase 3 enrollment for HLP003 in depression; data due Q4 2026. Compass is on track for COMP360's NDA, and an ayahuasca ingredient showed promise in a phase 2a trial.
The FDA moved to exclude semaglutide, tirzepatide and liraglutide from its 503B compounding list. Eli Lilly won U.S. approval for its oral pill Foundayo, and Novo Nordisk sued Lilly over allegedly misleading weight-loss ads.
