Oral dersimelagon clears Phase III bar in ultra-rare EPP and XLP

Japanese drugmaker Tanabe Pharma said its oral candidate dersimelagon met the primary endpoint and showed a favorable safety profile in INSPIRE, a global Phase III trial in erythropoietic protoporphyria and X-linked protoporphyria, two ultra-rare disorders marked by painful reactions to light.

The conditions carry a significant day-to-day burden of strict light avoidance for patients and families.

The study compared once-daily dersimelagon 200 mg with placebo for 16 weeks in adults and adolescents, followed by an ongoing open-label extension. Tanabe said the primary endpoint was met, based on time to first prodromal symptoms after sunlight exposure.