March 29, 2024, Shanghai, China – Alebund Pharmaceuticals (“Alebund” or the “Company”), an integrated biopharmaceutical company focusing on developing innovative therapies for the treatment of renal diseases and related chronic conditions, announced today that the US Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to AP303 for the treatment of autosomal dominant polycystic kidney disease (ADPKD).
“We are very excited that AP303 has been granted Orphan Drug Designation by the FDA”, Dr. Gavin Xia, Co-founder, Chairman, and CEO of Alebund commented. “It is an important milestone for Alebund as well as our efforts to address the significant unmet medical needs with ADPKD. We look forward to advancing AP303 to improve future treatment options for patients in need.”
AP303, a novel drug candidate developed in-house by Alebund, has demonstrated a meaningful improvement of renal survival in an ADPKD mice model. AP303 has completed its first-in-human study in healthy subjects in Australia and is ready for a Phase II trial in ADPKD. The prevalence of ADPKD is reported to be between 1 in 400 and 1 in 1,000 live births; it is an important cause of end-stage kidney disease (ESKD) requiring renal replacement therapy.1 This Orphan Drug Designation reflects the need for more new treatment options and the potential for AP303 to address the pressing needs of people living with ADPKD.
The US FDA’s ODD is a special status granted to support the development and evaluation of potential new medicines intended for the treatment, diagnosis, or prevention of rare diseases or disorders that affect fewer than 200,000 people in the US. The designation provides incentives to advance the development of treatments for rare diseases, including marketing exclusivity, tax credits for clinical research costs, and exemption from prescription drug user fees.
Reference
- Bergmann, C., Guay-Woodford, L.M., Harris, P.C. et al. Polycystic kidney disease. Nat Rev Dis Primers 4, 50 (2018). https://doi.org/10.1038/s41572-018-0047-y
About ADPKD
ADPKD is the most common monogenic kidney disease and one of the leading causes of end-stage kidney disease in adults worldwide. Its course is characterized by the development and inexorable expansion of multiple cysts scattered throughout the kidney parenchyma. ADPKD is most caused by mutations in the genes PKD1 (in 78% of disease pedigrees) or PKD2 (in 15% of disease pedigrees). Treatment recommendation for patients with ADPKD includes anti-hypertensive treatment, dietary restrictions, and pharmacotherapy for a limited percentage of suitable patients. More broadly applicable treatment options with fewer side effects are needed to effectively slow the decline of kidney function and improve kidney outcomes in patients with ADPKD.
About Alebund Pharmaceuticals
Alebund is a biopharmaceutical company jointly incubated by a group of industry leaders in the field of nephrology in Shanghai in 2018. Alebund focuses on the discovery and development of novel therapies primarily for kidney diseases and their complications, as well as other chronic conditions. Alebund has built a diversified and balanced pipeline of drug candidates targeting a range of renal diseases, including chronic kidney disease (CKD)/dialysis complications, IgA nephropathy, diabetic kidney disease, and autosomal dominant polycystic kidney disease (ADPKD). Alebund’s pipeline comprises both small-molecule and biological assets, in which many programs are in the clinical stage.
礼邦医药在研创新药物 AP303 获得美国食品药品监督管理局（FDA）孤儿药资格认定, 用于治疗常染色体显性多囊肾病（ADPKD）
2024 年 3 月 29 日，中国上海—礼邦医药（“礼邦”或“公司”），一家专注于开发治疗肾脏疾病及相关慢性病创新药物的综合性生物制药公司，宣布其在研创新药物 AP303 获得美国食品药品监督管理局（FDA）孤儿药资格认定，用于治疗常染色体显性多囊肾病（ADPKD）。
“我们非常高兴 AP303 被 FDA 授予孤儿药资格。”礼邦联合创始人、董事长兼首席执行官夏国尧博士评论道，“这对于礼邦医药以及我们为解决 ADPKD 疾病未满足的临床需求所做的努力而言都是一个重要里程碑。我们会努力推进 AP303 项目，为 ADPKD 患者提供更多的治疗选择。”
AP303 是礼邦医药自行研发的创新药物，在 ADPKD 小鼠模型中观察到肾脏存活率提高，并已完成其在健康受试者中的首次人体试验，准备进行 ADPKD Ⅱ 期临床研究。ADPKD 在新生儿中的患病率约为四百分之一到千分之一；该疾病是导致终末期肾病（ESKD）需接受肾脏替代治疗的重要原因。1此次孤儿药认定显示出治疗 ADPKD 的迫切临床需求，以及 AP303 未来为患者提供更多治疗手段的潜力。
孤儿药资格认定是 FDA 对符合条件的用于预防、治疗及诊断罕见病的药物授予的一种资格认定，旨在支持治疗、诊断或预防影响美国不到 200,000 人的罕见疾病或病症的潜在新药的开发和评估。这样的资格认定为促进罕见疾病治疗的开发提供了激励措施，包括在药物上市的市场独占权、临床研究费用税收减免以及免除上市申请费用等。
参考文献
- Bergmann, C., Guay-Woodford, L.M., Harris, P.C. et al. Polycystic kidney disease. Nat Rev Dis Primers 4, 50 (2018). https://doi.org/10.1038/s41572-018-0047-y
关于常染色体显性多囊肾病（ADPKD）
ADPKD 是最常见的单基因肾病，也是全球导致成人终末期肾病的主要原因之一。其主要表现为肾脏囊肿不断增大、增多，破坏正常的肾脏结构和功能。ADPKD 最常见的原因是多囊肾致病基因 PKD1（在 78% 的疾病谱系中）或 PKD2（在 15% 的疾病谱系中）突变。对于 ADPKD 患者的治疗建议包括抗高血压治疗、饮食限制以及仅适合部分患者的药物治疗。ADPKD 患者迫切需要可治疗更多患者且副作用更少的有效治疗药物以减缓患者的肾功能衰退并改善肾脏结局。
关于礼邦医药
2018 年初，礼邦医药由顶尖的肾脏病领域行业领导者孵化于中国上海，是一家处于临床阶段的生物制药公司，主要致力于肾脏病以及其他相关慢性疾病创新药物的发现和开发，为慢性肾脏病及相关疾病患者提供更佳临床治疗方案。礼邦医药已经建立起了丰富且均衡的肾脏病新药产品管线，包括针对慢性肾病（CKD）/透析并发症、IgA 肾病、糖尿病肾病、常染色体显性多囊肾病（ADPKD）等产品，公司在研产品包括小分子药物和生物制剂，目前多个项目处于临床试验阶段。