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Pacritinib

Enpaxiq

Extended indication Patients with myelofibrosis who have thrombocytopenia
Therapeutic value No judgement
Total cost 945,000.00
Registration phase Registration application pending

Product

Active substance Pacritinib
Domain Oncology and Hematology
Reason of inclusion New medicine (specialité)
Main indication Other hematology
Extended indication Patients with myelofibrosis who have thrombocytopenia
Proprietary name Enpaxiq
Route of administration Oral
Therapeutical formulation Capsule
Budgetting framework Intermural (MSZ)
Additional remarks Tyrosine kinase inhibitor gericht tegen JAK2 en FLT3

Registration

Registration route Centralised (EMA)
Type of trajectory Normal trajectory
Submission date July 2017
Expected Registration February 2019
Orphan drug Yes
Registration phase Registration application pending
Additional remarks Pacritinib was al eerder ingediend in juni 2016 maar werd teruggetrokken in maart 2017 vanwege tijdtekort om nieuwe data in de aanvraag te includeren.

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

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 remarks 118 diagnoses in 2016. Trombocytopenie komt voor bij 18-28% van de myelofibrosis patiënten (21-33 patiënten).

Expected cost per patient per year

Cost 30,000.00 - 40,000.00
References GIPdatabank
Additional remarks De vergoeding per patiënt in 2015 voor de huidige behandelmethode met ruxolitinib was €32.174,00.

Potential total cost per year

Total cost

945,000.00

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.

Off label use

There is currently nothing known about off label use.

Indication extension

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

Other information

There is currently no futher information available.