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Venetoclax

Venclyxto

Extended indication Venetoclax in combination with low dose cytarabine or in combination with a hypomethylating agent is
Therapeutic value Possible added value
Total cost 2,800,000.00
Registration phase Clinical trials

Product

Active substance Venetoclax
Domain Oncology and Hematology
Reason of inclusion Indication extension
Main indication AML / MDS
Extended indication Venetoclax in combination with low dose cytarabine or in combination with a hypomethylating agent is indicated for the treatment-naive patients with AML who are ineligible for intensive chemotherapy.
Proprietary name Venclyxto
Manufacturer Abbvie
Mechanism of action Bcl-2 inhibitor
Route of administration Oral
Therapeutical formulation Tablet
Budgetting framework Intermural (MSZ)
Additional remarks Apoptosis stimulant; proto-oncogene protein c-bcl-2 inhibitor.

Registration

Registration route Centralised (EMA)
Submission date 2019
Expected Registration May 2020
Orphan drug Yes
Registration phase Clinical trials
Additional remarks Breakthrough-therapy designation granted by the FDA. Venetoclax is in de sluis geplaatst.

Therapeutic value

Current treatment options EMA: Treatment for AML is complex and depends on a number of factors including the extent of the disease, whether it has been treated before, and the patient’s age, symptoms and general state of health. At the time of designation, the main treatments for AML were chemotherapy (medicines to treat cancer) and haematopoietic (blood) stem-cell transplantation (a complex procedure where the patient receives stem cells from a matched donor to help restore the bone marrow).
Therapeutic value Possible added value
Substantiation EMA: The sponsor has provided sufficient information to show that venetoclax might be of significant benefit for patients with AML because early studies show that, when used in combination with other medicines, it can produce a response in the disease in patients older than 65 years who cannot receive standard treatment. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.
Duration of treatment Median 7 month / months
Frequency of administration 1 times a day
Dosage per administration 400/600 mg
References FDA venetoclax highlights of prescribing information (versie 11/2018).
Additional remarks Lijkt zeer veelbelovende combinatie; respons rates van 70-80%. Middel wordt binnenkort getest, in combinatie met intensieve chemotherapie, in HOVON studie. Documentatie van de FDA geeft aan dat de "median duration of exposure" voor patiënten die venetoclax gebruikten in combinatie met azacitidine of decitabine gelijk is aan respectievelijk 6,5 maanden en 8,4 maanden. De "median duration of exposure" voor patiënten die venetoclax gebruikten in combinatie met een lage dosis cytarabine was 3,9 maanden. Inschatting: behandelduur van 7 maanden (mediaan).

Expected patient volume per year

Patient volume

20 - 120

Market share is generally not included unless otherwise stated.
References NKR; CBS
Additional remarks NKR 2015: 674. Clinical trial in patienten >65; CBS: percentage bevolking >65 jaar; 17,8%. Veel patiënten ontvangen chemotherapie. Om die reden verwachten we dat het realistisch patiëntvolume nog lager zal liggen.

Expected cost per patient per year

Cost 40,000.00
References Medicijnkosten.nl, fabrikant, FDA
Additional remarks Meerkosten. Met een AIP van €53,37 per tablet van 100 mg. Een dosering van 400 mg per dag en €5711 per cyclus (van 28 dagen) met een opbouwschema startend met 100 mg op dag 1, 200 mg op dag 2 en 300 mg op dag 3, komt de behandeling van AML met venetoclax per patiënt met zo'n 7 cycli neer op in totaal €40.000,00. Venetoclax is in de sluis geplaatst om te komen tot een financieel arrangement.

Potential total cost per year

Total cost

2,800,000.00

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
References Clinicaltrials.gov, SPS.
Additional remarks Er lopen meerdere fase III studies met Venetoclax maar niet in combinatie met cytarabine.

Other information

There is currently no futher information available.