olezarsen sodium 获欧盟孤儿药资格认定后遭撤回，用于治疗家族性乳糜微粒血症综合征
Orphan designation: olezarsen sodium Treatment of familial chylomicronaemia syndrome, 21/08/2024 Withdrawn
olezarsen sodium 于 2024 年 8 月 21 日获欧盟孤儿药资格认定，用于治疗家族性乳糜微粒血症综合征，该认定现已被撤回。孤儿药资格认定使申办方获得 EMA 科学与监管支持以推进至上市许可申请阶段，但不代表药物已获批上市。该认定编号为 EU/3/24/2973。
Overview
This medicine was designated as an orphan medicine for the treatment of familial chylomicronemia syndrome in the European Union on 21 August 2024.
This means that the developer will receive scientific and regulatory support from EMA to advance their medicine to the stage where they can apply for a marketing authorisation.
Orphan designation does not mean the medicine is available or authorised for use. All medicines, including designated orphan medicines, must be authorised before they can be marketed and made available to patients in the EU.
During the medicine's development, doctors may be able to enrol patients in clinical trials investigating the medicine. For information on ongoing clinical trials in the EU, see:
Key facts
Active substance
olezarsen sodium
Intended use
Treatment of familial chylomicronaemia syndrome
Orphan designation status
Withdrawn
EU designation number
EU/3/24/2973
Date of designation
21/08/2024
Sponsor
来源：EMA What's New · ema.europa.eu