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Omaveloxolone

Extended indication Treatment of Friedreich's Ataxia
Therapeutic value Possible added value
Registration phase Registration application pending

Product

Active substance Omaveloxolone
Domain Neurological disorders
Reason of inclusion New medicine (specialité)
Main indication Muscular diseases other
Extended indication Treatment of Friedreich's Ataxia
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 Registration application pending

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

References Drugs.com
Additional remarks De prijs in de Verenigde Staten is $32,477 voor 90 capsules. De prijs in de Verenigde Staten ligt vaak hoger dan de Nederlandse prijs.

Potential total cost per year

There is currently nothing known about the possible total cost.

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.