PC is a chronically debilitating rare genetic disease resulting in lifelong limited mobility and severe pain. There are no FDA approved therapies. Our phase 3 clinical study of QTORIN™ rapamycin 3.9% for people with PC is currently enrolling. Palvella has been awarded both FDA Orphan Drug and Fast Track designation for this program.
Gorlin syndrome is a serious rare genetic disease where people may develop thousands of basal cell carcinomas, a form of cancerous skin tumors. There are no FDA approved therapies for Gorlin syndrome. Palvella is conducting a phase 2b clinical study of QTORIN™ rapamycin 3.9% for people with Gorlin syndrome.
Microcystic Lymphatic Malformations (Micro LM) is a rare genetic disease of the lymphatic system characterized by abnormal vessels or cysts that leads to chronic lymphorrhea, bleeding, pain, and a risk of severe life-threatening infections. There are no FDA approved therapies. Palvella is conducting a phase 2 clinical study for QTORIN™ rapamycin 3.9%, for people with this disease.