Omaveloxolone

Extended indication	Skyclarys is indicated for the treatment of Friedreich’s ataxia in adults and adolescents aged 16 years and older.
Therapeutic value	Possible added value
Total cost	116,620,000.00
Registration phase	Registered

Product

Active substance	Omaveloxolone
Domain	Neurological disorders
Reason of inclusion	New medicine (specialité)
Main indication	Muscular diseases other
Extended indication	Skyclarys is indicated for the treatment of Friedreich’s ataxia in adults and adolescents aged 16 years and older.
Proprietary name	Skyclarys
Manufacturer	Reata
Route of administration	Oral
Therapeutical formulation	Capsule
Budgetting framework	Extramural (GVS)
Additional remarks	Nrf2 activator

Registration

Registration route	Centralised (EMA)
Type of trajectory	Normal trajectory
ATMP	No
Submission date	December 2022
Expected Registration	February 2024
Orphan drug	Yes
Registration phase	Registered
Additional remarks	Geregistreerd februari 2024

Therapeutic value

Current treatment options	Revalidatie, geen echte behandeloptie.
Therapeutic value	Possible added value
Substantiation	Er is op dit moment nog geen directe behandeling mogelijk. De fase 2 studie is gepubliceerd in 2021 (1). Omaveloxolone verbeterde significant de neurologische functies vergeleken met placebo en was veilig. Recent is er ook een extensie-studie gepubliceerd. Deze resultaten bevestigen de positieve resultaten uit de MOXIe fase 2 en laten een voordeel zien van omaveloxolone behandeling gemeten in mFARS.
Duration of treatment	continuous
Frequency of administration	1 times a day
Dosage per administration	2,5-300 mg
References	NCT02255435; Ann Neurol. 2021 Feb;89(2):212-225 (1); Mov Disord. 2023 Feb;38(2):313-320 Efficacy of Omaveloxolone in Friedreich's Ataxia;

Expected patient volume per year

Patient volume	< 340 Market share is generally not included unless otherwise stated.
References	Spierziekten.nl (1)
Additional remarks	Friedreich's ataxia komt voor bij 1 op de 50.000 mensen (1). Voor Nederland zou dit dan gaan om 340 mogelijke patiënten.

Patient volume

< 340

Market share is generally not included unless otherwise stated.

References

Spierziekten.nl (1)

Additional remarks

Friedreich's ataxia komt voor bij 1 op de 50.000 mensen (1). Voor Nederland zou dit dan gaan om 340 mogelijke patiënten.

Expected cost per patient per year

Cost	343,000.00
References	McKenzie H. FDA approves first therapy for Friedreich’s ataxia (updated). BioSpace. 1 March 2023. Accessed 9 March 2023. https://www.biospace.com/article/fda-approvesfirst-therapy-for-friedreich-s-ataxia/
Additional remarks	De prijs in de Verenigde Staten is $32,477 voor 90 capsules. De prijs per patiënt per jaar wordt $370.000. De exacte prijs in Nederland is nog niet bekend.

Cost

343,000.00

References

McKenzie H. FDA approves first therapy for 
Friedreich’s ataxia (updated). BioSpace. 1 March 
2023. Accessed 9 March 2023. 
https://www.biospace.com/article/fda-approvesfirst-therapy-for-friedreich-s-ataxia/

Additional remarks

De prijs in de Verenigde Staten is $32,477 voor 90 capsules. De prijs per patiënt per jaar wordt $370.000. De exacte prijs in Nederland is nog niet bekend.

Potential total cost per year

Total cost	116,620,000.00 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.

Total cost

116,620,000.00

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	Yes
Indication extensions	Mitochondrial disorders; Ocular inflammation; Ocular pain; Malignant melanoma
References	Adis insight
Additional remarks	Huidige studies bevinden zich in fase 2.

Other information

There is currently no futher information available.