Lukasz Jarzyna, Vice President of Rare Disease at LEO Pharma, discusses the Priority Review for dersimelagon in the treatment of erythropoietic protoporphyria (EPP) and X-linked protoporphyria (XLP).
EPP and XLP are rare congenital metabolic disorders characterized by an inborn error of porphyrin-heme biosynthesis. Signs and symptoms include painful cutaneous photosensitivity that results in tingling, burning, pain, and itching within minutes of sun/light exposure and may be accompanied by swelling and redness. Other symptoms may include erythrodontia, red discoloration of urine, hemolytic anemia, and splenomegaly.
On September 18, the US Food and Drug Administration (FDA) announced Priority Review to the New Drug Application (NDA) for dersimelagon for the treatment of EPP and XLP, with a PDUFA date of February 2027. Dersimelagon is an investigational, once-daily oral small-molecule MC1R agonist being developed for EPP and XLP. Under a Priority Review designation, the FDA aims to take action on an application within 6 months, compared to 10 months under standard review. LEO Pharma also announced an acquisition of worldwide rights to dersimelagon from Tanabe Pharma.
Earlier this year, Tanabe Pharma announced results from the global, randomized, double-blind, placebo-controlled phase 3 INSPIRE clinical trial (NCT06144840). Dersimelagon demonstrated statistically significant and clinically meaningful outcomes across primary and secondary endpoints, including a significant prolongation of average daily sunlight exposure time to first prodromal symptoms.
If approved, dersimelagon could become the first oral treatment for EPP and XLP. Dersimelagon has not yet been approved by the FDA or any other regulatory authority, and its safety and efficacy have not been established by any regulatory authority.
For more information, visit https://leo-pharma.com/media-center/news/leo-pharma-announces-fda-acceptance-of-dersimelagon-nda-with-priority-review-and-closes-acquisition-from-tanabe-pharma/
To learn more about EPP and XLP and other rare skin conditions, visit https://checkrare.com/diseases/skin-conditions/