Palvella的QTORIN 3.9%雷帕霉素凝胶在3期微囊淋巴管畸形试验中显示出强大的疗效和安全性，95%的患者在第24周时改善

2026-02-24 19:38

Primary endpoint met with statistically significant improvement (mean change of +2.13; p<0.001) on the Microcystic Lymphatic Malformation Investigator Global Assessment (mLM-IGA)
Achieved statistical significance on pre-specified key secondary endpoint (p<0.001) and all four secondary efficacy endpoints (all p<0.001)
95% of trial participants aged ≥ 6 who completed the efficacy evaluation period improved on the mLM-IGA at Week 24
86% of trial participants aged ≥ 6 who completed the efficacy evaluation period were rated as "Much Improved" (+2) or "Very Much Improved" (+3) on the mLM-IGA at Week 24
QTORIN™ rapamycin was well-tolerated, with no drug-related serious adverse events reported and systemic rapamycin levels below 2ng/mL at all timepoints for all participants
98% of participants who completed the efficacy evaluation period elected to continue to receive QTORIN™ rapamycin in the ongoing treatment extension period
Palvella plans to submit a New Drug Application to FDA in the second half of 2026
QTORIN™ rapamycin has the potential to become the first FDA-approved therapy and standard of care for the estimated more than 30,000 individuals with microcystic lymphatic malformations in the U.S.