再鼎医药宣布,美国食品药品监督管理局(FDA)已授予ZL-1310孤儿药资格认定;ZL-1310是一款高活性的具有同类首创潜力的DLL3 ADC,用于治疗小细胞肺癌。 孤儿药(Orphan Drug)是指用于预防、治疗、诊断罕见病的药物。罕见病是指发病率极低的疾病,通常在人群中的发病率低于1/2000或更低。 ZL-1310将有资格获得某些开发激励...
Orphan DrugsPhenylketonuriaUnited StatesThere has never been a drug used to treat phenylketonuria (PKU), a genetic disorder caused by a deficiency of the enzyme phenylalanine hydroxylase (PAH), until now. Last month...
The aptamer, originally developed to treat osteogenesis imperfecta, has been granted Orphan Drug Designation and Pediatric Rare Disease Designation by the US Food and Drug Administration (FDA).XLH ("X" denotes X-chromosome, a sex-determining chromosome) is a ...
” said Richard Pazdur, M.D., director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research. “These improvements have been observed in chronic and accelerated
值得注意的是,表1仅仅总结了FDA在2022年批准的新药中的孤儿药。实际上,有很多药物再利用 (drug repurposing) 的开发都针对罕见病,他们获批的孤儿药并不在表1中体现。 例如Eton Pharmaceuticals/Azurity Pharmaceuticals公司2022年获批的治疗癫痫的唑尼沙胺口服...
The US Food and Drug Administration (FDA) has granted orphan drug designation for Arcturus Therapeutics’ ARCT-032 for cystic fibrosis.
Friedreich's Ataxia Research Alliance (FARA) has announced in a press release that the FDA has granted orphan drug status to Edison Pharmaceutical’s EPI-743 (vatiquinone). The drug treats Friedreich’s ataxia, a rare nervous system disorder. Guy Miller, MD, PhD, Chairman & CEO, Edison ...
The FDA has granted an orphan drug designation to FORE8394 for the treatment of primary brain and central nervous system malignancies. Stacie Shepherd, MD, PhD The FDA has granted an orphan drug designation to FORE8394 for the treatment of primary brain and central nervous system (CN...
Orphan drug designation 罕见病患者人数少,无法通过药物销售回收研发成本,这导致药企没有动力开发罕见病药物,由此也带来大量未被满足的临床需求——到20 世纪 80 年代,美国有 20-2500 万公民患有5,000 种罕见疾病。 鉴于此,美国国会于 1983 年推出《孤儿药法案》(ODA),提出一系列推动罕见病药物研发的政策,如,税...
Adverse effects reported in patients who received the treatment included pancreatitis, anaphylaxis, blood clotting, hemorrhaging, abnormal transaminases and bilirubin, hyperglycemia and nausea. For more information please visit theFDA website.