Extended indication

Symkevi is indicated in a combination regimen with ivacaftor 150 mg tablets for the treatment of pat

Therapeutic value

Possible added value

Total cost

6,475,000.00

Registration phase

Registered

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 150 mg 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

Additional comments
Positieve CHMP-opinie in september 2020.

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

30 - 40

Market share is generally not included unless otherwise stated.

References
Nederlands Cyctic Fibrosis Stichting; GVS-advies lumacaftor/ivacaftor (Orkambi®) voor cystische fibrose patiënten van 6 tot en met 11 jaar
Additional comments
Dit betreft patiënten naast de groep met een dubbele F508del-mutatie die op dit moment Orkambi gebruikt. Het aantal van 30-40 patiënten is een extrapolatie van de ingeschatte verhouding tussen Symkevi en Orkambi bij patiënten >12 jaar (140:498) en het aantal ingeschat voor Orkambi binnen deze leeftijdsgroep (124).

Expected cost per patient per year

Cost

185,000.00

References
Fabrikant

Potential total cost per year

Total cost

6,475,000.00

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.