Expand all sections

Omaveloxolone

Skyclarys

Extended indication Skyclarys is indicated for the treatment of Friedreich’s ataxia in adults and adolescents aged 16 ye
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.

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 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.

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.