Pacritinib

Active substance
Pacritinib
Domain
Oncology and Hematology
Reason of inclusion in Horizonscan Geneesmiddelen
New medicine (specialité)
Main indication
Myeloproliferative disorders
Extended indication
Patients with myelofibrosis who have thrombocytopenia

1. Product

Proprietary name
Epjevy
Manufacturer
Cti
Mechanism of action
JAK tyrosine kinase inhibitor
Route of administration
Oral
Therapeutical formulation
Capsule
Budgetting framework
Intermural (MSZ)
Additional comments
Tyrosine kinase inhibitor gericht tegen JAK2 en FLT3

2. Registration

Registration route
Centralised (EMA)
Type of trajectory
Normal trajectory
Submission date
July 2017
Orphan drug
Yes
Registration phase
Withdrawn
Additional comments
Pacritinib was al eerder ingediend in juni 2016 maar werd teruggetrokken in maart 2017 vanwege tijdtekort om nieuwe data in de aanvraag te includeren. De aanvraag die in juli 2017 is ingediend is in februari 2019 teruggetrokken met als reden dat CTI niet is staat is data te leveren binnen de procedurele tijdslijn om de vragen van de EMA te adresseren.

3. Therapeutic value

Current treatment options
Ruxolitinib
Therapeutic value
No judgement
Duration of treatment
Median 15.6 month / months
Frequency of administration
1 times a day
Dosage per administration
400 mg

4. Expected patient volume per year

Patient volume

21 - 33

Market share is generally not included unless otherwise stated.

References
NKR; Mesa et al. Lancet Haematol. 2017 May;4(5):e225-e236.
Additional comments
118 diagnoses in 2016. Trombocytopenie komt voor bij 18-28% van de myelofibrosis patiënten (21-33 patiënten).

5. Expected cost per patient per year

Cost
30,000 - 40,000
References
GIPdatabank
Additional comments
De vergoeding per patiënt in 2015 voor de huidige behandelmethode met ruxolitinib was €32.174,00.

6. Potential total cost per year

Total cost

945,000

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

Indication extension
No
Additional comments
geen fase III studies voor andere indicaties dan in de horizonscan

9. Other information

There is currently no futher information available.