Sarizotan Medicine 13 December 2018 Extended indication Rett syndrome with respiratory symptoms Therapeutic value No judgement Total cost Registration phase Clinical trials Product Active substance Sarizotan Domain Lung diseases Reason of inclusion New medicine (specialité) Main indication Lung other Extended indication Rett syndrome with respiratory symptoms Current proprietary name Already available biosimilars / generics Proprietary name Manufacturer Newron Portfolio holder Mechanism of action Unknown Route of administration Oral Therapeutical formulation Capsule Budgetting framework Extramural (GVS) Centre of expertise Additional remarks A serotonin (5-HT)1A receptor agonist and a partial agonist/antagonist at D2 receptor. Registration Registration route Centralised (EMA) Type of trajectory Unknown Particularity Unknown ATMP Unknown Submission date 2019 Expected Registration 2020 Orphan drug Yes Registration phase Clinical trials Reimbursement Medicine sluice Additional remarks Therapeutic value Current treatment options geen Therapeutic value No judgement This assessment does not indicate any potential inclusion in the package. Substantiation Duration of treatment Frequency of administration 1 times a day Dosage per administration 2 - 10 mg References NCT02790034 Additional remarks Expected patient volume per year Patient volume < 250 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References De Nederlandse Rett Syndroom Vereniging (NRSV) Additional remarks Het Rett syndroom heeft een incidentie van 1 op de 10.000 tot 23.000 geboren meisjes, het is extreem zeldzaam bij jongens. Het is beschreven bij alle rassen en etnische groepen. In Nederland zijn op dit moment ruim 250 meisjes met het Rett syndroom bekend. 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 No Indication extensions References Additional remarks Other information Additional remarks