Sarizotan Medicine 13 December 2018 Extended indication Rett syndrome with respiratory symptoms Therapeutic value No judgement Total cost Registration phase No registration expected 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 Receptor agonist Route of administration Oral Therapeutical formulation Capsule Budgetting framework Extramural (GVS) Centre of expertise Additional remarks Sarizotan is een serotonine (5-HT)1A receptor agonist en een partial agonist/antagonist van de D2 receptor. De ademhalingsregulatiestoornissen bij Rett syndroom worden gerelateerd aan functionele/neurotransmitter stoornissen in de (hersenstam)kernen die de ademhaling aansturen. Registration Registration route Centralised (EMA) Type of trajectory Unknown Particularity Unknown ATMP Unknown Submission date Expected Registration Orphan drug Yes Registration phase No registration expected Reimbursement Medicine sluice Additional remarks Klinische studies stopgezet 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 150 - 175 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. In Nederland zijn op dit moment ruim 250 meisjes met het Rett syndroom bekend. Ongeveer 60%-70% van deze patiënten heeft last van ademhalingsstoornissen (150-175). 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