QTORIN rapamycin

QTORIN rapamycin is a novel, patented 3.9% anhydrous gel formulation of rapamycin (also known as sirolimus), developed for topical administration. It is designed to selectively inhibit the mammalian target of rapamycin (mTOR) pathway in the skin, thereby reducing endothelial cell hyperproliferation and vascular endothelial growth factor signaling—key drivers in diseases such as microcystic lymphatic malformations (LMs) and cutaneous venous malformations. The formulation aims to deliver high concentrations of active drug directly to affected tissue while minimizing systemic absorption and associated adverse effects seen with oral or systemic mTOR inhibitors. Developed by Palvella Therapeutics, QTORIN rapamycin has received Breakthrough Therapy Designation, Fast Track Designation, and Orphan Drug Designation from the FDA for microcystic LMs; it is also being investigated for other serious genetic skin diseases driven by overactivation of the mTOR pathway[1][2][5][6][8].

Brand names

QTORIN

Other names

sirolimus