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Givinostat

Extended indication Ambulant Patients With Duchenne Muscular Dystrophy
Therapeutic value Possible added value
Registration phase Registration application pending

Product

Active substance Givinostat
Domain Neurological disorders
Reason of inclusion New medicine (specialité)
Main indication Duchenne
Extended indication Ambulant Patients With Duchenne Muscular Dystrophy
Manufacturer Italfarmaco
Route of administration Oral
Therapeutical formulation Oral suspension
Budgetting framework Extramural (GVS)
Additional remarks Givinostat is een histon deacetylase (HDAC) inhibitor.

Registration

Registration route Centralised (EMA)
Type of trajectory Normal trajectory
ATMP No
Submission date August 2023
Expected Registration September 2024
Orphan drug Yes
Registration phase Registration application pending

Therapeutic value

Therapeutic value Possible added value
Substantiation De primaire eindpunten van de EPIDYS trial zijn gehaald.
Duration of treatment continuous
Frequency of administration 2 times a day
References NCT02851797

Expected patient volume per year

Patient volume

< 500

Market share is generally not included unless otherwise stated.
References Spierziekten Nederland; Clemens et al. JAMA Neurol. 2020;77(8):982-991 (1). NCT02851797 (2)
Additional remarks Er zijn in Nederland rond de 500 patiënten met Duchenne spierdystrofie (1). De groep waarop getest wordt in de klinische studies (ambulante patiënten van 6 jaar of ouder) betreft een deel van deze populatie (2).

Expected cost per patient per year

References Drugs.com
Additional remarks In de Verenigde Staten zijn de kosten voor Farydak orale capsule 10 mg bedragen ongeveer $ 14.838 voor 6 capsules. De prijs in de Verenigde Staten is doorgaans hoger dan in Nederland.

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 Myeloproliferative disorders, Polycythaemia vera
References Adis insight

Other information

There is currently no futher information available.