Cabaletta Bio 的 CABA-201 获得 FDA 的孤儿药资格认定,这是一种潜在的肌炎治疗方法。 Cabaletta Bio receives Orphan Drug Designation for CABA-201, a potential myositis treatment, from the FDA.
临床阶段生物技术公司 Cabaletta Bio 宣布,其 CABA-201 已获得美国食品和药物管理局的孤儿药资格认定,CABA-201 是一种治疗特发性炎症性肌病(也称为肌炎)的潜在疗法。 Cabaletta Bio, a clinical-stage biotechnology company, announced it has received orphan drug designation from the Food and Drug Administration for CABA-201, a potential treatment for idiopathic inflammatory myopathies, also known as myositis. 该称号是为了表彰开发旨在治疗罕见疾病的疗法,突显了治疗肌炎方面未得到满足的医疗需求,肌炎是一种严重且可能致命的自身免疫性疾病,目前尚无治疗方法。 This designation is awarded for developing therapies aimed at treating rare diseases, highlighting the unmet medical need in treating myositis, a severe and potentially fatal autoimmune disease for which no curative therapy currently exists.