Multiple Acyl Coenzyme A Dehydrogenase Deficiency
IntraBio’s Levacetylleucine Succeeds in Phase 3 Trial for Ataxia-Telangiectasia, Plans FDA and EMA Approval
IntraBio; levacetylleucine; Ataxia-Telangiectasia; Phase 3; FDA approval; EMA; rare disease
CHMP Rejects Sanofi’s Rezurock Over Clinical Data Issues, Recommends Approval of BTK Inhibitor Wayrilz
Sanofi; CHMP; EMA; Rezurock; Wayrilz; Belumosudil; Rilzabrutinib; chronic graft-versus-host disease; cGVHD; immune thrombocytopenia; ITP; regulatory approval; clinical data
Rezon Bio Launches Two State-of-the-Art CDMO Facilities for Biologics in Europe
Rezon Bio; biologics; CDMO; contract manufacturing; Gdańsk; Warsaw-Duchnice; Europe; digital innovation; GMP manufacturing; biosimilars; EMA; FDA
CHMP Recommends Against Approval of Elevidys Gene Therapy in Europe, Dealing Blow to Sarepta and Roche
CHMP; Elevidys; Sarepta; Roche; gene therapy; Duchenne muscular dystrophy; EMA; negative opinion; Europe; EMBARK trial
CHMP Recommends Five New Drugs and Reviews Eli Lilly’s Kisunla
CHMP; EMA; new drug approvals; Eli Lilly; Kisunla; biosimilars; hyperphosphatemia; myopia; March 2025; regulatory review
EMA Identifies Very Rare Eye Condition as Side Effect of Semaglutide
EMA; semaglutide; rare side effect; NAION; vision loss; Ozempic; Wegovy; Rybelsus
EMA Reverses Decision, Recommends Approval of Lecanemab (Leqembi) for Early Alzheimer’s Disease in Specific Patient Group
Lecanemab, Leqembi, Alzheimer’s disease, EMA, CHMP, early Alzheimer’s disease, ApoE ε4, amyloid-beta monoclonal antibody
European Commission’s Reconsideration of Translarna’s Marketing Authorization: A Critical Turning Point for Duchenne Muscular Dystrophy Treatment
Translarna, Duchenne muscular dystrophy, European Commission, EMA, CHMP, conditional marketing authorization, drug approval, regulatory decisions
European Medicines Agency Investigates Potential Link Between Hair Loss Drugs and Suicidal Thoughts
EMA, hair loss drugs, finasteride, dutasteride, suicidal thoughts, psychiatric side effects, European Medicines Agency
FDA Delays Dupixent Decision for COPD by Three Months, EMA Recommends Approval
FDA, Dupixent, COPD, EMA, Regeneron, Sanofi, Priority Review, PDUFA, Boreas, Notus, Phase III Trials