Dersimelagon Medicine 04 December 2025 Extended indication Dersimelagon monotherapy for treatment of X-linked or erythropoietic protoporphyria in adolescents, adults and elderly. Therapeutic value Total cost Registration phase Clinical trials Product Active substance Dersimelagon Domain Metabolism and Endocrinology Reason of inclusion New medicine (specialité) Main indication Metabolic diseases Extended indication Dersimelagon monotherapy for treatment of X-linked or erythropoietic protoporphyria in adolescents, adults and elderly. Current proprietary name Already available biosimilars / generics Proprietary name Manufacturer Mitsubishi Tanabe Portfolio holder Mitsubishi Tanabe Mechanism of action Route of administration Oral Therapeutical formulation Tablet Budgetting framework Intermural (MSZ) Centre of expertise Additional remarks Registration Registration route Centralised (EMA) Type of trajectory Normal trajectory Particularity ATMP No Submission date July 2026 Expected Registration August 2027 Orphan drug Yes Registration phase Clinical trials Reimbursement Medicine sluice Additional remarks Therapeutic value Current treatment options Therapeutic value Substantiation Duration of treatment Frequency of administration Dosage per administration References NCT06144840 (INSPIRE) Additional remarks Expected patient volume per year Patient volume Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References Additional remarks Expected cost per patient per year Cost References Additional remarks Potential total cost per year Total cost Total cost for sluice Additional remarks Off label use Off label use Indications off label use References Additional remarks Indication extension Indication extensions Indication extensions References Additional remarks Other information Additional remarks