Conclusions
XLP, Treatment-related
EPP
In this first-in-human phase 1 study, dersimelagon was generally well tolerated following oral administration and demonstrated a generally consistent PK profile across diverse groups of healthy participants. An effective and safe orally administered treatment will address an important unmet need for patients with EPP and XLP, especially for pediatric and adolescent patients who have had no approved pharmacological options to date. Treatment-related increases in melanin density following multiple doses of dersimelagon support further investigation into the photoprotective effects of dersimelagon. A recently completed phase 3 study (