Sarizotan

Active substance
Sarizotan
Domain
Lung diseases
Reason of inclusion in Horizonscan Geneesmiddelen
New medicine (specialité)
Main indication
Other lung
Extended indication
Rett syndrome with respiratory symptoms

1. Product

Manufacturer
Newron
Mechanism of action
Receptor agonist
Route of administration
Oral
Therapeutical formulation
Capsule
Budgetting framework
Extramural (GVS)
Additional comments
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.

2. Registration

Registration route
Centralised (EMA)
Submission date
2020
Expected Registration
2021
Orphan drug
Yes
Registration phase
Clinical trials

3. Therapeutic value

Current treatment options
geen
Therapeutic value
No judgement
Duration of treatment
Not found
Frequency of administration
1 times a day
Dosage per administration
2 - 10 mg
References
NCT02790034

4. 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 comments
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).

5. Expected cost per patient per year

There is currently nothing known about the expected cost.

6. Potential total cost per year

There is currently nothing known about the possible total cost.

7. Off label use

There is currently nothing known about off label use.

8. Indication extension

Indication extension
No

9. Other information

There is currently no futher information available.