sirolimus (Nobelpharma Co., Ltd.)

Sirolimus (also known as rapamycin) is a potent small molecule inhibitor of the mechanistic target of rapamycin (mTOR) pathway. NPC-12G is a topical gel formulation of sirolimus developed by Nobelpharma specifically for the treatment of skin lesions associated with vascular abnormalities and genetic conditions such as tuberous sclerosis complex (TSC). The drug works by binding to the intracellular protein FKBP12; the resulting complex then binds to and inhibits mTOR Complex 1 (mTORC1). This inhibition suppresses the overactive mTOR signaling that drives abnormal cell proliferation and angiogenesis in vascular lesions, leading to the regression of angiofibromas and other skin manifestations. While a version of this gel is approved in Japan for TSC-related skin lesions, NPC-12G is being evaluated in multi-regional Phase 3 trials for broader vascular abnormality-associated skin lesions.

Brand names

Rapalimus

Other names

rapamycinRapalimus Gel