Palvella's QTORIN 3.9% Rapamycin Gel Demonstrates Strong Efficacy And Safety In Phase 3 Microcystic Lymphatic Malformations Trial, 95% Patients Improved At Week 24
Benzinga Newsdesk 8 months ago
News
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
- 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.