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 remarks
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 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

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 remarks
Huidige studies bevinden zich in fase 2.

Other information

There is currently no futher information available.