WAYNE, PA — NFlection Therapeutics, Inc. recently announced that the Food and Drug Administration (FDA) has granted the company orphan drug designation for NFX-179 for the treatment of cutaneous neurofibromatosis type 1. NFX-179 is a topical, first-in-class, “soft” MEK inhibitor that is currently being evaluated in Phase 2 clinical trials in patients with cutaneous neurofibromatosis type 1.
The FDA’s Office of Orphan Drug Products grants orphan status to support development of medicines for underserved patient populations, or rare disorders, that affect fewer than 200,000 people in the United States. Orphan drug designation provides to NFlection certain benefits, including market exclusivity upon regulatory approval if received, exemption of FDA application fees and tax credits for qualified clinical trials.
The prevalence of cutaneous neurofibromatosis type 1 is estimated to be between 100,000 and 120,000 in the United States. Cutaneous neurofibromas are tumors that grow from small nerves in the skin or just under the skin and appear as small or larger bumps typically beginning around the time of puberty. Individuals commonly develop more cutaneous neurofibromas as they get older. These may be disfiguring, itchy or painful when bumped. Despite their benign nature, they may cause significant problems (e.g., depression, isolation, etc.) and may require surgical removal.
“Receiving orphan drug designation for NFX-179 is another important milestone in the development of NFX-179 Gel for the treatment of cutaneous neurofibromatosis,” said Christopher Powala, President and Chief Executive Officer of NFlection. “We are pleased with the progress we are making in the clinic and believe NFX-179 Gel is an important novel therapy for the treatment of RASopathies, such as cutaneous neurofibromatosis, and we look forward to progressing NFX-179 Gel to Phase 2b.”
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