Immix Biopharma Awarded European Union Orphan Drug Designation For NXC-201 In AL Amyloidosis
Immix Biopharma Awarded European Union Orphan Drug Designation For NXC-201 In AL Amyloidosis
European Orphan Drug Designation ("ODD") qualifies NXC-201 for:
欧洲孤儿药称号（“ODD”）使 NXC-201 有资格获得：
- 10 years of market exclusivity once authorized in the EU
- Access to the EU centralized authorization procedure
- Reduced fees for EU protocol assistance, marketing authorization applications, inspections before authorization, applications for changes to marketing authorizations made after approval, and reduced annual fees
- 一旦获得欧盟授权，市场独家经营权将持续10年
- 进入欧盟集中授权程序
- 降低了欧盟协议援助、上市许可申请、授权前检查、批准后提出的上市许可变更申请的费用，并降低了年费
U.S. observed prevalence of relapsed/refractory AL Amyloidosis is growing 12% per year according to Staron, et al Blood Cancer Journal, estimated to reach 29,712 patients in 2023
根据Staron等人《血癌杂志》，美国观察到的复发/难治性AL淀粉样变患病率每年增长12％，估计到2023年将达到29,712名患者
Los Angeles, Feb. 07, 2024 (GLOBE NEWSWIRE) -- Immix Biopharma, Inc. (("Immix Biopharma", "Company", "We" or "Us", NASDAQ:IMMX), a clinical-stage biopharmaceutical company trailblazing cell therapies in autoimmune disease, today announced that the European Commission (EC) has granted orphan drug designation to NXC-201 for the treatment of AL Amyloidosis.
洛杉矶，2024 年 2 月 7 日（GLOBE NEWSWIRE）—— 开创自身免疫性疾病细胞疗法的临床阶段生物制药公司 Immix Biopharma, Inc.（“Immix Biopharma”、“公司”、“我们” 或 “我们”，纳斯达克股票代码：IMMX）今天宣布，欧盟委员会（EC）已将 NXC-201 的孤儿药认定为用于治疗 AL 淀粉样变性。
"European Union orphan drug designation for NXC-201 represents an important milestone in our global strategy," said Ilya Rachman, MD PhD, Chief Executive Officer, Immix Biopharma. "We believe we are addressing an urgent need to establish a new potential one-time treatment option for relapsed/refractory AL Amyloidosis patients." Gabriel Morris, Chief Financial Officer, Immix Biopharma, added, "We believe NXC-201's observed favorable tolerability profile and 'Single Day CRS' across a robust clinical dataset to-date enables our observed complete responses (ongoing) in heavily pretreated relapsed/refractory AL Amyloidosis patients and potential expansion into other autoimmune diseases."
Immix Biopharma首席执行官伊利亚·拉赫曼博士说：“欧盟认定 NXC-201 孤儿药是我们全球战略中的一个重要里程碑。”“我们认为，我们正在解决为复发/难治性AL淀粉样变患者建立新的潜在一次性治疗选择的迫切需求。”Immix Biopharma首席财务官加布里埃尔·莫里斯补充说：“我们认为，迄今为止，在强大的临床数据集中，NXC-201 观察到的良好的耐受性状况和'单日CRS'使我们在经过大量预处理的复发/难治性 AL 淀粉样变患者中观察到完全的反应（正在进行中），并有可能扩展到其他自身免疫性疾病。”
Orphan drug designation in the European Union (EU) is granted by the EC based on a positive opinion issued by the European Medicines Agency (EMA) Committee for Orphan Medicinal Products. To qualify for orphan drug designation, a candidate therapy must be intended for the treatment, prevention or diagnosis of a life-threatening or chronically debilitating disease that occurs in not more than five in 10,000 people in the EU. The designation provides regulatory, financial and commercial incentives to develop therapies for rare diseases where there are either no satisfactory treatment options or significant benefit to those affected by the disease.
欧盟（EU）的孤儿药认证是由欧盟根据欧洲药品管理局（EMA）孤儿药产品委员会发布的积极意见授予的。要获得孤儿药认定的资格，候选疗法必须用于治疗、预防或诊断一种危及生命或慢性衰弱的疾病，这种疾病发生在欧盟每10,000人中不超过5人。该称号为开发罕见疾病疗法提供了监管、财政和商业激励措施，这些疾病既没有令人满意的治疗选择，也没有对受该疾病影响的人带来重大益处。