FDA approved Imaavy (nipocalimab-aahu), the first treatment for warm autoimmune hemolytic anemia; 24% achieved durable hemoglobin responses vs 8% with placebo. Rilzabrutinib earned Breakthrough status.
Gene therapies for Fabry and Gaucher disease showed sustained benefits in early trials: all 11 Fabry patients stopped enzyme replacement therapy, and four of six Gaucher patients stayed off standard treatment for up to two years.
Two new studies in IBD genetics show that polygenic risk scores can predict disease severity in Danish patients, and that ancestry-specific variants in Hispanic populations reveal novel risk loci and differential effects of known genes. These findings advance precision medicine by improving risk prediction and highlighting the need for diverse genetic research.
Multiple studies confirm high effectiveness of key antimalarial drugs in Indonesia, Ethiopia, and for malaria prevention in HIV-positive pregnancies. Research shows DHA-PPQ, artemether-lumefantrine, and other treatments remain well-tolerated with no delayed parasite clearance. Findings support continued use of current treatment policies while emphasizing the need for ongoing monitoring.
Disc Medicine has agreed with the FDA to resubmit bitopertin for rare blood disorder erythropoietic protoporphyria, with the agency allowing the Phase 3 APOLLO trial to support a new filing. The agreement follows a February rejection and could lead to a mid-2027 decision. Disc has implemented workforce cuts and its CFO sold shares amid the regulatory setback.
A study of over 1.1 million births found a U-shaped relationship between early pregnancy hemoglobin levels and severe neonatal morbidity and mortality, with both anemia and erythrocytosis increasing risk.
Ruxolitinib remains the standard frontline therapy for intermediate-2 and high-risk myelofibrosis, with phase 3 and real-world data showing spleen and symptom benefits. Cytopenias, transfusion needs and resistance after 2 to 3 years continue to drive interest in new combinations.
A study of 2,282 adults aged 60 and older found anemia was associated with higher Alzheimer’s disease blood biomarkers and a 66% higher chance of developing dementia. The highest risk was seen in participants with both anemia and higher biomarker levels.
Final phase 3 trial results show atrasentan (Vanrafia®) provides significant long-term kidney function benefits in IgA nephropathy patients, with a 2.59 mL/min/1.73m² eGFR improvement versus placebo. Earlier phase 2 data demonstrated the drug reduces proteinuria by 30.7% when added to standard background therapy. The drug's manufacturer plans to seek traditional FDA approval in 2026 based on these findings.
The FDA has approved Lifyorli (relacorilant) plus nab-paclitaxel for platinum-resistant ovarian cancer based on trial data showing improved survival. Separately, the agency granted Breakthrough Therapy designation to Lilly's sofetabart mipitecan for the same condition, expediting development of the antibody-drug conjugate.
