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:
歐洲孤兒藥稱號（“ODD”）使 NXC-201 有資格獲得：
- 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
- 一旦獲得歐盟授權，市場獨家經營權將持續10年
- 進入歐盟集中授權程序
- 降低了歐盟協議援助、上市許可申請、授權前檢查、批准後提出的上市許可變更申請的費用，並降低了年費
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
根據Staron等人《血癌雜誌》，美國觀察到的復發/難治性AL澱粉樣變患病率每年增長12％，估計到2023年將達到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.
洛杉磯，2024 年 2 月 7 日（GLOBE NEWSWIRE）—— 開創自身免疫性疾病細胞療法的臨床階段生物製藥公司 Immix Biopharma, Inc.（“Immix Biopharma”、“公司”、“我們” 或 “我們”，納斯達克股票代碼：IMMX）今天宣佈，歐盟委員會（EC）已將 NXC-201 的孤兒藥認定爲用於治療 AL 澱粉樣變性。
"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首席執行官伊利亞·拉赫曼博士說：“歐盟認定 NXC-201 孤兒藥是我們全球戰略中的一個重要里程碑。”“我們認爲，我們正在解決爲復發/難治性AL澱粉樣變患者建立新的潛在一次性治療選擇的迫切需求。”Immix Biopharma首席財務官加布裏埃爾·莫里斯補充說：“我們認爲，迄今爲止，在強大的臨床數據集中，NXC-201 觀察到的良好的耐受性狀況和'單日CRS'使我們在經過大量預處理的復發/難治性 AL 澱粉樣變患者中觀察到完全的反應（正在進行中），並有可能擴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.
歐盟（EU）的孤兒藥認證是由歐盟根據歐洲藥品管理局（EMA）孤兒藥產品委員會發布的積極意見授予的。要獲得孤兒藥認定的資格，候選療法必須用於治療、預防或診斷一種危及生命或慢性衰弱的疾病，這種疾病發生在歐盟每10,000人中不超過5人。該稱號爲開發罕見疾病療法提供了監管、財政和商業激勵措施，這些疾病既沒有令人滿意的治療選擇，也沒有對受該疾病影響的人帶來重大益處。
譯文內容由第三人軟體翻譯。