LEO Pharma Acquires Potential First Oral Therapy for EPP and XLP
Key Takeaways
- LEO Pharma’s acquisition of dersimelagon expands its rare dermatology footprint alongside recent moves involving spesolimab for GPP and a herpes simplex virus gene therapy platform.
- Phase 3 INSPIRE (NCT06144840), a global randomized double-blind placebo-controlled study, achieved statistically significant, clinically meaningful primary and secondary outcomes supporting the June 2026 NDA.
LEO Pharma will acquire worldwide rights to dersimelagon, an investigational oral therapy under FDA review for EPP and XLP.
Dersimelagon has completed phase 3 development, and a new drug application (NDA) for EPP and XLP was submitted to the US Food and Drug Administration (FDA) in June 2026. If approved, it would become the first oral therapy for EPP and XLP.1
The acquisition builds on LEO Pharma's expansion into
Phase 3 INSPIRE Trial Supports Dersimelagon NDA
Dersimelagon was evaluated in the
Results announced in March 2026 showed dersimelagon met the study's primary and secondary end points, with statistically significant and clinically meaningful outcomes. The findings were also presented as a late-breaking presentation at the
Among the reported functional outcomes, dersimelagon significantly prolonged average daily sunlight exposure time to first prodromal symptoms. Exact effect sizes and P values were not provided in the LEO Pharma announcement.
Dersimelagon is designed to increase melanin in the skin, helping reduce sunlight penetration and protect against phototoxic reactions.
FDA Review Underway
Dersimelagon has received FDA fast track and orphan drug designations for EPP and XLP. An NDA for both indications was submitted to the FDA in June 2026. The therapy has not been approved by the FDA or another regulatory agency, and its safety and efficacy have not been established by a regulatory authority.1
The acquisition could also mark a broader shift in the treatment landscape for EPP and XLP, where emerging therapies are beginning to expand options for patients with rare phototoxic disorders.
"The dermatology rare disease space is poised for another milestone victory, placing the oral melanocortin-1 receptor agonist, dersimelagon, into the care of LEO Pharma, a company with vested interest in rare dermatology diseases given their recent acquisition of spesolimab for GPP. For EPP/XLP porphyria patients, dersimelagon is a therapy that can deliver a meaningful improvement in patient symptoms and quality of life based on phase 3 data. It joins a list of emerging innovations for EPP, marking a turning point for care of EPP/XLP patients," Christopher G. Bunick, MD, PhD, editor in chief of Dermatology Times, told the publication.
Dersimelagon is a therapy that can deliver a meaningful improvement in patient symptoms and quality of life — Christopher G. Bunick, M.D., Ph.D.
Under the acquisition agreement, LEO Pharma will acquire worldwide rights to dersimelagon for up to $435 million in upfront and near-term milestone payments, along with potential downstream milestones and tiered royalties on net sales.¹
References
- LEO Pharma further strengthens late-stage pipeline with the acquisition of dersimelagon. News release. LEO Pharma. August 18, 2026. Accessed August 18, 2026.
https://www.businesswire.com/news/home/20260817946702/en/LEO-Pharma-Further-Strengthens-Late-Stage-Pipeline-With-the-Acquisition-of-Dersimelagon - INcreased Sun Exposure Without Pain In Research Participants With EPP or XLP (INSPIRE). ClinicalTrials.gov. Identifier: NCT06144840. Accessed August 18, 2026.
https://clinicaltrials.gov/study/NCT06144840 - Tanabe Pharma announces positive data from phase 3 INSPIRE study of dersimelagon in EPP and XLP. News release. Tanabe Pharma Corporation. March 30, 2026.
https://www.tanabe-pharma.com/en/news/rel_260330/main/0/link/e_rel_260330.pdf
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