Palvella -Model QTORIN -Pachyonychia Congenita (PC)
PC is a rare, chronically debilitating and lifelong disease in which mutations of genes responsible for keratin production lead to dysregulated keratinocyte proliferation, increased skin fragility, and impaired skin barrier function on the feet. Affected individuals have difficulty walking, and often require ambulatory aids or crawling.
Palvella estimates that there are 9,300 people with PC diagnosed and treated by Dermatologists and Podiatrists in the US.
There are no FDA approved therapies for PC.
The potential for rapamycin in PC was discovered by leading scientists and geneticists in the field of PC research who demonstrated a direct mechanism of action of mTOR inhibitors on translationally repressing the mutant keratin genes which are the root cause of PC.
QTORINTM rapamycin 3.9% has been awarded Fast Track Designation, FDA Orphan Drug Designation, and EMA orphan drug designation for PC.