Regeneron's Garetosmab在第三阶段试验中将FOP患者的新骨骼生长减少了90%至94%。
Regeneron's garetosmab reduced new bone growth in FOP patients by 90% to 94% in Phase 3 trial.
Regenenron制药公司报告说,Garetosmab试验了第3阶段取得积极结果,Garetosmab是一种实验性药物,治疗骨骼纤维畸形增生(FOP),是一种罕见的遗传疾病,导致软组织骨骼异常生长。
Regeneron Pharmaceuticals reported positive Phase 3 trial results for garetosmab, an experimental drug treating fibrodysplasia ossificans progressiva (FOP), a rare genetic disorder causing abnormal bone growth in soft tissues.
与FOP成人安慰剂相比,新骨骼损伤减少了90%至94%。
The trial met its primary endpoint, showing a 90% to 94% reduction in new bone lesions compared to placebo in adults with FOP.
药物目标A是活性A, 一种与不正常骨骼形成有关的蛋白质。
The drug targets activin A, a protein linked to abnormal bone formation.
Regenenron计划到2025年底提交美国批准,并于2026年在全球提交,计划为较年轻的病人进行新的试验。
Regeneron plans to file for U.S. approval by year-end 2025 and submit globally in 2026, with a new trial planned for younger patients.
治疗方案影响到全世界大约900名被诊断的个人,Garetosmab可以成为一个关键的治疗方案,与目前核准的索霍诺斯药物竞争。
FOP affects about 900 diagnosed individuals worldwide, and garetosmab could become a key treatment option, competing with the currently approved drug Sohonos.