Expand all sections

Voretigene neparvovec

Luxturna

Extended indication RPE65-mediated inherited retinal disease (IRD).
Therapeutic value Possible added value
Total cost 5,300,000.00
Registration phase Registration application pending

Product

Active substance Voretigene neparvovec
Domain Neurological disorders
Main indication Eye disorders
Extended indication RPE65-mediated inherited retinal disease (IRD).
Proprietary name Luxturna
Manufacturer Spark
Route of administration Subretinal
Therapeutical formulation Injection
Budgetting framework Intermural (MSZ)
Additional remarks Werkingsmechanisme: Voretigene neparvovec is DNA dat codeert voor een functioneel RPE65 eiwit.

Registration

Registration route Centralised (EMA)
Type of trajectory Normal trajectory
Particularity New medicine
Submission date September 2017
Expected Registration October 2018
Orphan drug No
Registration phase Registration application pending

Therapeutic value

Current treatment options Geen
Therapeutic value Possible added value
Substantiation In de fase 3 trial konden patiënten met interventie iets beter het 'doolhof' door met weinig lichtintensiteit ten opzichte van controles (primair eindpunt). Secundair eindpunt: visuswinst 8 letters op de ETDRS kaart (FDA vond geen statistisch significante verschillen), Goldmann gezichtsveld iets beter, foveale gevoeligheid niet beter, QoL vragenlijst betere score ten opzichte van controles.
Dosage per administration 0,3 ml
References Russell et al. Lancet. 2017 Aug 26;390(10097):849-860; Voretigene Neparvovec for Biallelic RPE65- Mediated Retinal Disease: Effectiveness and Value. Draft Evidence Report November 14, 2017; ICER
Additional remarks Eenmalige subretinale injectie per oog. Duur van het effect is onbekend, lijkt nu 2-5 jaar aan te houden, daarna zijn er geen studiedata.

Expected patient volume per year

Patient volume

2 - 18

Market share is generally not included unless otherwise stated.
References EMA; vision2020
Additional remarks Patiëntaantallen in de VS worden geschat op 350 per jaar. Omgerekend in populatie zou dat voor Nederland 18 patiënten per jaar betekenen. Expert opinion zegt dat er waarschijnlijk maar 1-2 patiënten per jaar in aanmerking komen.

Expected cost per patient per year

Cost 400,000.00 - 660,000.00
References Scrip; Voretigene Neparvovec for Biallelic RPE65- Mediated Retinal Disease: Effectiveness and Value. Draft Evidence Report November 14, 2017; ICER
Additional remarks Jan 2018: $850.000 voor beide ogen, of $425.000 per oog.

Potential total cost per year

Total cost

5,300,000.00

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

There is currently nothing known about indication extensions.

Other information

There is currently no futher information available.