FREIBURG IM BREISGAU, Germany, July 03, 2024 / Biotech Newswire / -- Eleva, a pioneer in unlocking difficult-to-produce biologics based on a breakthrough manufacturing platform, announced today that the European Commission has granted the Orphan Drug Designation for Factor H (CPV-104), the company’s recombinant version of human complement Factor H, to treat C3 Glomerulopathy (C3G).

弗赖堡-布雷斯高，德国，2024年7月3日/Biotech Newswire/--Eleva是基于突破性制造平台解锁难以生产的生物制剂的先驱，今天宣布，欧盟委员会已授予孤儿药H因子（CPV-104）的名称，该公司的重组人补体因子H，用于治疗C3肾小球病（C3G）。

Eleva is preparing a first clinical study in C3G and expects to commence dosing patients in H1 2025.“Today’s news marks an important milestone for Eleva’s R&D and regulatory teams, as the Orphan Drug Designation adds significant value to the Factor H (CPV-104) program. The positive opinion by the EMA is also a clear validation for the comprehensive preclinical data sets we have generated for our program,” commented Andreas Schaaf, Ph.D., Chief Scientific Officer of Eleva.

Eleva正在准备C3G的第一项临床研究，预计将于2025年上半年开始给患者服用。“今天的新闻标志着Eleva研发和监管团队的一个重要里程碑，因为孤儿药名称为H因子（CPV-104）计划增加了重要价值。EMA的积极意见也清楚地验证了我们为我们的计划生成的综合临床前数据集，”Eleva首席科学官Andreas Schaaf博士评论道。

“Overall, Factor H (CPV-104) continues to progress steadily towards clinical studies and is emerging rapidly as a potential first-in-class solution for C3G and potentially other complement-related disorders.”The Orphan Drug Designation (ODD) in the EU was granted by the European Commission based on a positive opinion issued previously by the EMA Committee for Orphan Medicinal Products (COMP).

“总的来说，H因子（CPV-104）在临床研究方面继续稳步进展，并迅速成为C3G和潜在的其他补体相关疾病的潜在一流解决方案。”欧盟的孤儿药名称（ODD）是由欧盟委员会根据EMA孤儿药品委员会（COMP）先前发表的积极意见授予的。

The program is focused on the development of drugs that have shown the potential to provide significant benefit to patients suffering from rare, life-threatening diseases. Following market approval, the ODS designation would provide Eleva with ten years of market exclusivity and provides special incentives, including eligibility for protocol assistance and possible exemptions or reductions in certain regulatory fees.C3G represents a rare renal disease caused by the dysregulation of the complement system, a central part of the body’s imm.

该计划的重点是开发药物，这些药物已显示出为患有罕见，威胁生命的疾病的患者提供显着益处的潜力。在获得市场批准后，ODS的指定将为Eleva提供十年的市场排他性，并提供特殊的激励措施，包括获得议定书援助的资格以及某些监管费用的可能豁免或减少。C3G代表一种罕见的肾脏疾病，由补体系统失调引起，补体系统是人体imm的核心部分。