Extended indication

Atgam is indicated for use in adults and in children aged 2 years and older for the treatment of acq

Therapeutic value

No estimate possible yet

Registration phase

Registration application pending

Product

Active substance

Horse anti-human T lymphocyte immunoglobulin

Domain

Cardiovascular diseases

Reason of inclusion

New medicine (specialité)

Main indication

Other non-oncological hematological medications

Extended indication

Atgam is indicated for use in adults and in children aged 2 years and older for the treatment of acquired moderate to severe aplastic anaemia of known or suspected immunologic aetiology as part of standard immunosuppressive therapy in patients who are unsuitable for bone marrow transplant.

Proprietary name

ATgam

Manufacturer

Pfizer

Mechanism of action

Immunostimulation

Route of administration

Intravenous

Therapeutical formulation

Concentrate for solution for infusion

Budgetting framework

Intermural (MSZ)

Centre of expertise

LUMC

Additional comments
ATGAM bestaat uit een mix van polyclonale antistoffen die verkregen wordt door immunisatie van paarden.

Registration

Registration route

Decentralised

Type of trajectory

Normal trajectory

ATMP

No

Submission date

January 2020

Expected Registration

March 2022

Orphan drug

No

Registration phase

Registration application pending

Therapeutic value

Current treatment options

Geen vergelijkbare behandelopties voor >80% van de patienten met aplastische anemie. Voor een klein deel van de patiënten met aplastische anemie tot 40 jaar kan allogene stamceltransplantatie overwogen worden bij aanwezigheid van een gematchte sibling donor.

Therapeutic value

No estimate possible yet

Frequency of administration

1 times a day

Dosage per administration

16 mg/kg/day over 10 days or 20 mg/kg/day over 8 days or 40 mg/kg/day over 4 days

References
Richtlijn verworven aplastische anemie, maart 2019: https://hematologienederland.nl/wp-content/uploads/2019/07/richtlijn_verworven_aplastische_anemie_2019_definitief.pdf ; Scheinberg P, et al. N EngI J Med 2011;365:430-8;
Additional comments
Treatment duration: 4 to 10 days (dependent on dose per day)
Total recommended dose is 160mg/kg

Expected patient volume per year

Patient volume

28

Market share is generally not included unless otherwise stated.

References
Nederlandse Aplastische Anemie register; Tjon JM, et al. Br J Haematol 2018;180:459-62.

Expected cost per patient per year

Additional comments
De fabrikant geeft aan dat de prijsstelling niet voor het eerste kwartaal van 2022 beschikbaar zal zijn.

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

No

Other information

There is currently no futher information available.