Tyra Biosciences 的药物 TYRA-300 获得 FDA 的软骨发育不全罕见儿科疾病认定。 Tyra Biosciences' drug TYRA-300 receives Rare Pediatric Disease Designation for achondroplasia from the FDA.
Tyra Biosciences 宣布，TYRA-300 已获得美国食品和药物管理局 (FDA) 的罕见儿科疾病认定，可作为软骨发育不全（最常见的侏儒症）的潜在治疗方法。 Tyra Biosciences announced it has received Rare Pediatric Disease Designation from the U.S. Food and Drug Administration (FDA) for TYRA-300 as a potential treatment for achondroplasia, the most common form of dwarfism. 该公司计划于 2024 年下半年向 FDA 提交研究性新药申请，进行一项 2 期临床试验，评估 TYRA-300 多剂量组治疗软骨发育不全儿童的效果。 The company plans to submit an Investigational New Drug application to the FDA in the second half of 2024 for a Phase 2 clinical trial evaluating multiple dose cohorts of TYRA-300 for children with achondroplasia. RPD 指定旨在加快罕见儿科疾病潜在治疗方法的开发和审查。 The RPD designation aims to expedite the development and review of potential treatments for rare pediatric diseases.