Imugene Limited has received a significant boost with the United States Food and Drug Administration (FDA) granting Orphan Drug Designation (ODD) to its experimental therapy, CF33-hNIS (VAXINIA), aimed at treating bile tract cancer. This designation propels Imugene’s clinical-stage oncolytic virotherapy forward, positioning the Sydney-based company as a key player in the development of innovative cancer […]
Telix Pharmaceuticals Limited (ASX: TLX) has taken a significant step in the realm of neuro-oncology by submitting a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for TLX101-CDx, also known as Pixclara (18F-floretyrosine or 18F-FET). This investigational positron emission tomography (PET) imaging agent is designed to enhance the characterization of progressive […]
Phanes Therapeutics, Inc., a clinical-stage biotechnology company dedicated to advancing oncology treatments, has achieved a significant milestone with the granting of Orphan Drug Designation (ODD) for its novel therapy, PT217. The U.S. Food and Drug Administration (FDA) awarded this status to PT217 for its potential in treating neuroendocrine carcinoma (NEC), a particularly aggressive form of […]
Alexion Pharmaceuticals has secured a significant milestone with the FDA granting priority review for its C5 complement inhibitor, SOLIRIS (eculizumab), as a treatment for neuromyelitis optica spectrum disorder (NMOSD) in patients who test positive for anti-aquaporin-4 (AQP4) autoantibodies. This critical approval comes as part of a supplemental Biologics License Application (sBLA), which has been accepted […]
Connecticut-based Biohaven Pharmaceutical has received a significant regulatory boost, with the US FDA granting orphan drug designation for its investigational drug verdiperstat for treating multiple system atrophy (MSA). This rare and debilitating neurodegenerative disease has long been a target for pharmaceutical companies seeking breakthroughs in treatment, and verdiperstat’s designation underscores its potential as a game-changer […]
British specialty biopharma company Mereo BioPharma Group has completed patient enrollment for a Phase 2b clinical trial of its human monoclonal antibody BPS-804, also known as Setrusumab. This trial aims to evaluate BPS-804 as a potential treatment for osteogenesis imperfecta (OI), commonly referred to as brittle bone disease. Osteogenesis imperfecta is a rare genetic disorder […]
