This author has yet to write their bio.Meanwhile lets just say that we are proud old admin contributed a whooping 84 entries.
MODERATE IMPACT – The FDA placed a clinical hold on the FORTIS phase 1/2 trial of AT845 following a case of peripheral sensory neuropathy in one trial participant. FORTIS is evaluating AT845, an investigational AAV8-based gene replacement therapy in adults with late-onset Pompe disease To date, the serious adverse event (SAE) has been classified by […]
MODERATE IMPACT – After a second day of presentations and discussions, the FDA has voted unanimously to endorse betibeglogene autotemcel (beti-cel) for the treatment of patients with beta-thalassemia who require regular red blood cell (RBC) transfusions. Bluebird Bio presented a strong case for beti-cel in the treatment of beta-thal with emphasis on high transfusion-independent rate […]
HIGH IMPACT – After a full day of presentation and debates around the risk vs benefit of Bluebird Bio’s elivaldogene autotemcel (eli-cel) for the treatment of early active cerebral adrenoleukodystrophy (CALD), the FDA unanimously voted to endorse the gene therapy. Initial concerns including malignancy with development of MDS, insertional oncogenesis, efficacy benchmarking, and long-term durability […]
MODERATE IMPACT – Although no detailed data were provided, Alexion/AstraZeneca showed positive topline results for the phase 3 open-label CHAMPION-NMOSD trial for Ultomiris. Ultomiris achieved a statistically significant and clinically meaningful reduction in the risk of relapse in adults with AQP4+ NMOSD compared to the external placebo arm from the pivotal Soliris PREVENT clinical trial. […]
HIGH IMPACT – Protalix and Chiesi announce topline results of Phase III BALANCE trial evaluating the safety and efficacy of PRX-102 in Fabry. Study: Phase III BALANCE study is a 12-month, randomized, double-blind, study of 1 mg/kg PRX-102 administered every two weeks compared to agalsidase beta. 78 enrolled patients were previously treated with agalsidase beta […]
