Pegcetacoplan Medicine 06 June 2023 Extended indication Extension of indication to include treatment of adult patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) not previously treated with a complement inhibitor Therapeutic value No estimate possible yet Total cost Registration phase Registration application pending Product Active substance Pegcetacoplan Domain Cardiovascular diseases Reason of inclusion Indication extension IND Main indication Other non-oncological hematological medications Extended indication Extension of indication to include treatment of adult patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) not previously treated with a complement inhibitor Current proprietary name Already available biosimilars / generics Proprietary name Aspaveli Manufacturer Sobi Portfolio holder Mechanism of action Complement inhibitor Route of administration Unknown Therapeutical formulation Unknown Budgetting framework Extramural (GVS) Centre of expertise Additional remarks Registration Registration route Centralised (EMA) Type of trajectory Normal trajectory Particularity Unknown ATMP No Submission date April 2023 Expected Registration February 2024 Orphan drug Yes Registration phase Registration application pending Reimbursement Medicine sluice Additional remarks Therapeutic value Current treatment options Therapeutic value No estimate possible yet This assessment does not indicate any potential inclusion in the package. Substantiation Duration of treatment Frequency of administration Dosage per administration References 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 Unknown Indications off label use References Additional remarks Indication extension Indication extensions Unknown Indication extensions References Additional remarks Other information Additional remarks