Omaveloxolone Medicine 07 December 2022 Extended indication Treatment of Friedreich's Ataxia Therapeutic value Possible added value Total cost 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 Current proprietary name Already available biosimilars / generics Proprietary name Manufacturer Reata Portfolio holder Mechanism of action Unknown Route of administration Oral Therapeutical formulation Capsule Budgetting framework Extramural (GVS) Centre of expertise Additional remarks Nrf2 activator Registration Registration route Centralised (EMA) Type of trajectory Normal trajectory Particularity Unknown ATMP No Submission date December 2022 Expected Registration October 2023 Orphan drug Yes Registration phase Registration application pending Reimbursement Medicine sluice Additional remarks Therapeutic value Current treatment options Revalidatie, geen echte behandeloptie. Therapeutic value Possible added value This assessment does not indicate any potential inclusion in the package. 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 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; Additional remarks Expected patient volume per year Patient volume < 340 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice 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 References Additional remarks Potential total cost per year Total cost Total cost for sluice Additional remarks Off label use Off label use Unknown Indications off label use References Additional remarks Indication extension Indication extensions 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 Additional remarks