Givinostat Medicine 07 December 2022 Extended indication Ambulant Patients With Duchenne Muscular Dystrophy Therapeutic value Possible added value Total cost 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 Current proprietary name Already available biosimilars / generics Proprietary name Manufacturer Italfarmaco Portfolio holder Mechanism of action Unknown Route of administration Oral Therapeutical formulation Oral suspension Budgetting framework Extramural (GVS) Centre of expertise Additional remarks Givinostat is een histon deacetylase (HDAC) inhibitor. Registration Registration route Centralised (EMA) Type of trajectory Normal trajectory Particularity Unknown ATMP No Submission date August 2023 Expected Registration January 2025 Orphan drug Yes Registration phase Registration application pending Reimbursement Medicine sluice Additional remarks Therapeutic value Current treatment options Therapeutic value Possible added value This assessment does not indicate any potential inclusion in the package. Substantiation De primaire eindpunten van de EPIDYS trial zijn gehaald. Duration of treatment Frequency of administration 2 times a day Dosage per administration References NCT02851797 Additional remarks Expected patient volume per year Patient volume < 500 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice 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 Cost References Drugs.com Additional remarks In de Verenigde Staten zijn de kosten voor Farydak orale capsule 10mg bedragen ongeveer $14,838 voor zes capsules. De prijs in de Verenigde Staten is doorgaans hoger dan in Nederland. 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 Myeloproliferative disorders, Polycythaemia vera References Adis insight Additional remarks Other information Additional remarks