topical rapamycin

Topical rapamycin (sirolimus) is a small molecule mTOR inhibitor developed by Dermatology Specialties Limited Partnership (DSLP) for the treatment of facial angiofibromas (FA) associated with Tuberous Sclerosis Complex (TSC). TSC is a genetic disorder caused by mutations in the TSC1 or TSC2 genes, which normally encode proteins that regulate the mechanistic target of rapamycin (mTOR) pathway. Loss of these tumor suppressors leads to constitutive mTOR activation, resulting in the development of hamartomatous skin lesions. This stabilized cream formulation (tested at 0.5% and 1.0% concentrations) is applied once daily to the affected areas. By inhibiting the mTORC1 complex, the drug suppresses the cellular proliferation and angiogenesis driving the growth of angiofibromas. Clinical data from Phase 2/3 trials indicate significant improvement in lesion appearance with minimal to no systemic absorption.

Other names

sirolimus creamrapamycin creamtopical sirolimus