Capricor Therapeutics Granted Orphan Drug And ATMP Status For Deramiocel By EMA
Capricor Therapeutics Granted Orphan Drug And ATMP Status For Deramiocel By EMA
Capricor Therapeutics (NASDAQ:CAPR), a biotechnology company developing transformative cell and exosome-based therapeutics for the treatment of rare diseases, today announced that the European Medicines Agency (EMA) has granted both Orphan Drug and Advanced Therapy Medicinal Product (ATMP) designations to its lead asset, deramiocel, for the treatment of Duchenne muscular dystrophy (DMD).
capricor therapeutics（納斯達克：CAPR）是一家開發基於電芯和外泌體治療稀有疾病的生物技術公司，今天宣佈歐洲藥品管理局（EMA）已將孤兒藥和愛文思控股藥品（ATMP）稱號授予其主要資產deramiocel，用於治療杜氏肌營養不良症（DMD）。
The Orphan Drug designation provides Capricor with several benefits that support the development of deramiocel in Europe, including market exclusivity for 10 years if approval is granted and substantially reduced regulatory fees. The ATMP designation provides substantial regulatory support to assist in the development of cell-based therapies. The additional support can potentially reduce time to market, streamline development, and open up access to critical resources, making it an invaluable tool for companies working on innovative therapies.
孤兒藥稱號爲capricor提供了多個支持deramiocel在歐洲開發的好處，包括在批准後擁有10年的市場獨佔權及大幅減少的監管費用。ATMP稱號則提供了 substantial監管支持，以協助開發基於電芯的療法。額外的支持可能會減少上市時間，簡化開發流程，並開放獲取關鍵資源，從而成爲致力於創新療法的公司的寶貴工具。