Extended indication Symkevi is indicated in a combination regimen with ivacaftor 150mg tablets for the treatment of pati
Therapeutic value Possible added value
Total cost 19,795,000.00
Registration phase Registered and reimbursed

Product

Active substance Tezacaftor / ivacaftor
Domain Lung diseases
Reason of inclusion Indication extension
Main indication Cystic fibrosis
Extended indication Symkevi is indicated in a combination regimen with ivacaftor 150mg tablets for the treatment of patients with cystic fibrosis (CF) aged 6 years and older who are homozygous for the F508del mutation or who are heterozygous for the F508del mutation and have one of the following mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene: P67L, R117C, L206W, R352Q, A455E, D579G, 711+3A→G, S945L, S977F, R1070W, D1152H, 2789+5G→A, 3272‑26A→G, and 3849+10kbC→T.
Proprietary name Symkevi
Manufacturer Vertex
Mechanism of action Protein chaperone
Route of administration Oral
Therapeutical formulation Tablet
Budgetting framework Extramural (GVS)
Centre of expertise UMCU, Haga Ziekenhuis, Erasmus MC, Amsterdam UMC, UMC St Radboud, UMCG, MUMC+
Additional comments In patiënten met F508del mutatie. CFTR modulation.

Registration

Registration route Centralised (EMA)
Type of trajectory Normal trajectory
Submission date December 2019
Expected Registration November 2020
Orphan drug Yes
Registration phase Registered and reimbursed
Additional comments Positieve CHMP-opinie in september 2020. Huidige bijlage 2 voorwaarde: uitsluitend in combinatie met ivacaftor voor de behandeling van cystische fibrose (CF) patiënten van 12 jaar en ouder die homozygoot zijn voor de F508del-mutatie in het CFTR-gen.

Therapeutic value

Current treatment options Geen vergelijkbare behandelopties bij deze leeftijdscategorie. Orkambi voor de homozygote F508del populatie.
Therapeutic value Possible added value
Substantiation Aangezien CF een progressieve ziekte betreft, heeft behandeling op jonge leeftijd extra meerwaarde gezien schade dan (gedeeltelijk) voorkomen kan worden.
Duration of treatment lifetime
Frequency of administration 2 times a day

Expected patient volume per year

Patient volume

< 107

Market share is generally not included unless otherwise stated.

References Data CF registratie 2019
Additional comments Er komen in deze leeftijdsgroep maximaal 107 patiënten in aanmerking voor behandeling.

Expected cost per patient per year

Cost 185,000.00
References Fabrikant

Potential total cost per year

Total cost

19,795,000.00

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

Off label use No
References Fabrikant: "Off label use risk to zero as product use and efficacy is related to CFTR mutation, for which NCFS registry data are exact estimate of prevalence"

Indication extension

Indication extension Yes
Indication extensions age extensions: - patients 2-5 yrs - patients 1-2 yrs - patients <1 years of age
References Fabrikant

Other information

There is currently no futher information available.