救济治疗报告称其新的液体沙普丁治疗对PKU是安全有效的,计划在2026年初提交给FDA. Relief Therapeutics reports its new liquid sapropterin therapy is safe and effective for PKU, with an FDA submission planned for early 2026.

救济治疗学宣布了一项关键研究的积极结果,该研究显示,其新的液体盐酸盐制剂(RLF-OD032)的生物当量相当于用于治疗苯丙酮尿(PKU)的KUVAN粉剂。 Relief Therapeutics has announced positive results from a pivotal study showing its new liquid sapropterin formulation, RLF-OD032, is bioequivalent to KUVAN® Powder for treating phenylketonuria (PKU). 这种即用型高浓度液体无需与水混合，可将剂量减少多达 100 倍，从而有可能提高依从性，尤其是儿童。 The ready-to-use, highly concentrated liquid requires no mixing with water and reduces dose volume by up to 100-fold, potentially improving adherence, especially in children. 这种治疗是完全容忍的,没有严重的副作用。 The treatment was well tolerated with no serious side effects. 该公司计划于2026年初向林业发展局提交一份505(b)(2)新的药物应用软件,寻求批准第一次便携、即时使用液体SApropterin疗法。 The company plans to submit a 505(b)(2) New Drug Application to the FDA in early 2026, seeking approval for the first portable, ready-to-use liquid sapropterin therapy. 如果获得批准,RLF-OD032可大大改善PKU病人的治疗便利和生活质量,预计至少2043年将获得专利保护。 If approved, RLF-OD032 could offer a major improvement in treatment convenience and quality of life for PKU patients, with patent protection expected through at least 2043.