Also known as: EMA
ACADIA Pharmaceuticals' stock jumped after the European Medicines Agency recommended approval of its Rett syndrome drug Daybue. The company also reported strong quarterly results and progress on its Alzheimer's disease psychosis treatment.
Regeneron announced FDA and EMA review acceptance for cemdisiran in generalized myasthenia gravis and FDA priority review acceptance for garetosmab in fibrodysplasia ossificans progressiva. The garetosmab BLA is supported by Phase 3 OPTIMA trial data showing 94% and 90% reductions in new bone lesions.
Acurx Pharmaceuticals is advancing ibezapolstat to international Phase 3 clinical trials for C. difficile infection following final regulatory guidance from FDA and EMA. The company launched a new clinical trial program for recurrent CDI and presented structural biology research demonstrating ibezapolstat's mechanism of action. Acurx reported year-end 2025 cash of $7.6 million.
The U.S. cardiovascular drugs market is projected to reach $37.03 billion by 2035, growing at a CAGR of 2.68%. Europe's anticoagulants market is expected to reach $23.68 billion by 2033, expanding at a CAGR of 8.22%.
AI-driven feasibility tools are cutting clinical trial site selection timelines from months to weeks by analysing historical data, enrolment benchmarks, and geographic trends. Clinical trial pharmacy software platforms are integrating with management systems and incorporating AI, machine learning, and blockchain to improve medication tracking, regulatory compliance, and data security across global trials.
Updated data from the phase I CaMMouflage trial showed CB-011, the first allogeneic anti-BCMA CAR-T therapy with immune cloaking, achieved an approximately 92% overall response rate in relapsed/refractory multiple myeloma. The 2026 Tandem Meetings also highlighted advances in EB-103, KITE-753, and LV20.19 CAR-T constructs across lymphoma and CLL. Separately, NXC-201 reported a 95% complete response rate in AL amyloidosis.
Sangamo Therapeutics has retained Raymond James to evaluate strategic alternatives to advance its pipeline and maximize stakeholder value. Key assets include the BLA-ready Fabry disease gene therapy ST-920, the STAC-BBB capsid platform generating $88M in fees to date, and multiple neurology programs. No transaction has been agreed and no timetable has been set.
Servier will acquire Edgewise Therapeutics' muscular dystrophy business, including the drug sevasemten, for up to $2.65 billion. Sevasemten recently faced an FDA rejection for accelerated approval in Becker muscular dystrophy but is advancing to a Phase 3 trial in 2026. The drug has received multiple FDA and EMA designations for both Becker and Duchenne muscular dystrophy.
Quantitative and functional MRI techniques are gaining traction as biomarkers in neurodegenerative diseases. A consensus statement provides MRI endpoint recommendations for clinical trials in hereditary ataxias like SCA and Friedreich ataxia. Separately, research shows resting-state fMRI can distinguish subtypes of multiple system atrophy and may improve early diagnosis.
The FDA has approved Shionogi's XOCOVA (ensitrelvir) as the first oral antiviral for post-exposure prophylaxis of COVID-19. Approval was based on the Phase 3 SCORPIO-PEP trial, which showed a 67% reduction in risk of symptomatic disease after exposure.
