Solid Biosciences宣布，其研发的SGT-212双路径基因疗法已获得美国食品药品监督管理局（FDA）授予的孤儿药认定，适应症为弗里德赖希共济失调。这一认定标志着该疗法在治疗罕见病领域迈出重要一步。
孤儿药认定旨在鼓励针对影响少数人群的罕见疾病开发创新疗法。获得该认定后，Solid Biosciences将在药物开发和商业化过程中享受一系列政策支持，包括临床试验费用税收抵免、豁免部分申请费用以及上市后七年的市场独占权。
弗里德赖希共济失调是一种遗传性神经系统疾病，目前尚无有效治疗方法。SGT-212通过双路径给药机制，有望为患者提供新的治疗选择。该公司表示将继续推进相关临床研究，加速疗法开发进程。
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