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Sarizotan

Extended indication Rett syndrome with respiratory symptoms
Therapeutic value No judgement
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
Manufacturer Newron
Mechanism of action Receptor agonist
Route of administration Oral
Therapeutical formulation Capsule
Budgetting framework Extramural (GVS)
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)
Orphan drug Yes
Registration phase No registration expected
Additional remarks Klinische studies stopgezet

Therapeutic value

Current treatment options geen
Therapeutic value No judgement
Frequency of administration 1 times a day
Dosage per administration 2 - 10 mg
References NCT02790034

Expected patient volume per year

Patient volume

150 - 175

Market share is generally not included unless otherwise stated.
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

There is currently nothing known about the expected cost.

Potential total cost per year

There is currently nothing known about the possible total cost.

Off label use

There is currently nothing known about off label use.

Indication extension

Indication extension No

Other information

There is currently no futher information available.