欧盟同意 nomlabofusp 治疗 Friedreich 共济失调的儿科研究计划
EMEA-003022-PIP01-21
欧盟就 nomlabofusp（重组融合蛋白，将人 frataxin 与 TAT 细胞穿透肽连接）用于治疗 Friedreich 共济失调的儿科研究计划作出同意决定，决定编号 EMEA-003022-PIP01-21，决定日期为 2025 年 7 月 10 日。该药拟以皮下给药方式使用，剂型为输注用溶液。
Key facts
Active substance
recombinant fusion protein linking human frataxin to TAT cell-penetrant peptide (nomlabofusp)
Therapeutic area
Congenital, familial and genetic disorders
Decision number
EMEA-003022-PIP01-21
PIP number
EMEA-003022-PIP01-21
Pharmaceutical form(s)
Solution for infusion
Condition(s) / indication(s)
Treatment of Friedreich's ataxia
Route(s) of administration
Subcutaneous use
Contact for public enquiries
Yes Pharmaceutical Development Services GmbH
Email: ra@larimartx.com
Tel: +1 (844)5119056Decision type
P: decision agreeing on a investigation plan, with or without partial waiver(s) and or deferral(s)
Decision date
10/07/2025
Compliance check done
No
来源：EMA What's New · ema.europa.eu