FDA Grants Priority Review to Dersimelagon for Rare Blood Disorders, Expediting Potential First Oral Treatment
The U.S. Food and Drug Administration (FDA) has granted Priority Review to the New Drug Application (NDA) for dersimelagon, an investigational, once-daily oral small-molecule MC1R agonist. This designation is for the treatment of erythropoietic protoporphyria (EPP) and X-linked protoporphyria (XLP), rare congenital metabolic disorders. EPP and XLP are characterized by painful cutaneous photosensitivity, leading to tingling, burning, pain, and itching upon sun or light exposure, often accompanied by swelling and redness. Other symptoms can include erythrodontia, red urine discoloration, hemolytic anemia, and splenomegaly. The FDA's Priority Review aims to take action on the application within six months, significantly faster than the standard ten-month review period. LEO Pharma, which recently acquired worldwide rights to dersimelagon from Tanabe Pharma, is developing the drug. Earlier this year, Tanabe Pharma announced positive results from the global, randomized, double-blind, placebo-controlled phase 3 I...
