NFlection Therapeutics Announces Orphan Drug Designation of NFX-179 for the Treatment of Cutaneous Neurofibromas

Author: BusinessWire
Published On: 09/01/2021

NFlection Therapeutics, Inc., a clinical-stage biopharmaceutical company focused on developing novel drug candidates for rare RASopathies, today 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.

“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.”

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.

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