Bedaquiline

Active substance
Bedaquiline
Domain
Infectious diseases
Reason of inclusion in Horizonscan Geneesmiddelen
Indication extension
Main indication
Bacterial infections
Extended indication
Sirturo is indicated for use as part of an appropriate combination regimen for pulmonary multidrug‑resistant tuberculosis (MDR‑TB) in adults and adolescent patients (12 years to less than 18 years of age and weighing at least 30 kg) when an effective treatment regimen cannot otherwise be composed for reasons of resistance or tolerability.

1. Product

Proprietary name
Sirturo
Manufacturer
Janssen
Mechanism of action
Enzyme inhibitor
Route of administration
Oral
Therapeutical formulation
Tablet
Budgetting framework
Intermural (MSZ)
Additional comments
ATP synthase-remmer

2. Registration

Registration route
Centralised (EMA)
Type of trajectory
Normal trajectory
Submission date
November 2018
Expected Registration
February 2020
Orphan drug
No
Registration phase
Registered and reimbursed
Additional comments
Positieve CHMP-opinie in december 2019. Geregistreerd in januari 2020.

3. Therapeutic value

Therapeutic value
Potential added value
Substantiation
Bij volwassenen behoort bedaquiline al tot de standaardzorg. Er wordt verwacht dat het geneesmiddel ook bij adolescenten een meerwaarde zal hebben.
Duration of treatment
Average 24 week / weeks
Frequency of administration
1 times a day
Dosage per administration
200-400 mg
Additional comments
De eerste twee weken ontvangt de patiënt 400mg eenmaal per dag, daarna 200mg driemaal per week.

4. Expected patient volume per year

Patient volume

1 - 3

Market share is generally not included unless otherwise stated.

References
Surveillance rapport RIVM geeft indicatie bij MDR / XDR TB.
Additional comments
Circa 5% van de TB patiënten zijn kinderen. Indicatie hoogstens 5 maal per jaar voor totale cohort (maximaal 1 kind per jaar). Recente WHO richtlijn wordt bedaquiline als belangrijkste aangemerkt gezien ontraden van injectables. In toekomst maximaal 15 per jaar geschat voor het totale cohort (dus maximaal 2-3 kinderen per jaar).

5. Expected cost per patient per year

Cost
21,734
References
G- standaard; SmPC; fabrikant
Additional comments
De huidige lijstprijs van 100mg, 188 stuks is € 21.734,61 exclusief BTW.

6. Potential total cost per year

Total cost

43,469

This amount gives an indication of the total cost. It is the result of the average expected patient volume times the average cost per patient. both per year.

7. Off label use

There is currently nothing known about off label use.

8. Indication extension

There is currently nothing known about indication extensions.

9. Other information

There is currently no futher information available.