Extended indication Friedreich's Ataxia
Therapeutic value No estimate possible yet
Registration phase Clinical trials

Product

Active substance Omaveloxolone
Domain Neurological disorders
Reason of inclusion New medicine (specialité)
Main indication Muscular diseases other
Extended indication Friedreich's Ataxia
Manufacturer Reata
Route of administration Oral
Therapeutical formulation Capsule
Budgetting framework Extramural (GVS)
Additional comments Nrf2 activator

Registration

Registration route Centralised (EMA)
Type of trajectory Normal trajectory
ATMP No
Submission date 2023
Expected Registration 2024
Orphan drug Yes
Registration phase Clinical trials

Therapeutic value

Current treatment options Revalidatie
Therapeutic value No estimate possible yet
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.
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);

Expected patient volume per year

Patient volume

< 340

Market share is generally not included unless otherwise stated.

References Spierziekten.nl (1)
Additional comments 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

There is currently nothing known about the expected cost.

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 comments Huidige studies bevinden zich in fase 2.

Other information

There is currently no futher information available.