Release Date: 21-Sep-2021
US FDA has granted orphan drug designation for NFX-179 for the treatment of cutaneous neurofibromatosis type 1. NFX-179 is a mitogen-activated protein kinase kinase (MEK) inhibitor. NFX-179 is a “soft” (metabolically labile) drug, that, when formulated as NFX-179 Gel for topical application, is intended to concentrate at the dermal site of action but reduce in systemic circulation, thereby expressively reducing side effects compared to systemically available MEK inhibitors. NFlection is evolving NFX-179 Gel for the treatment of RASopathies such as cutaneous neurofibromatosis type 1, immunosuppressant-mediated cutaneous squamous cell carcinoma, and congenital birthmarks.
The FDA's orphan-drug program gives special status to drugs and biologics for diseases and disorders that affect fewer than 200,000 people in the U.S and it also gives several benefits, like tax credits for qualified clinical trials, potential seven years of market exclusivity after approval and exemption from user fees.
Cutaneous neurofibromas are tumors that develop from small nerves in the skin or just under the skin and appear as small or larger bumps basically beginning at the time of puberty. These may be itchy, disfiguring or painful when bumped. They may cause several problems like depression, isolation, etc. and may require surgical removal. The prevalence of cutaneous neurofibromatosis type 1 is likely to be between 100,000 and 120,000 in the United States.