Horse anti-human T lymphocyte immunoglobulin

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

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

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 remarks	Treatment duration: 4 to 10 days (dependent on dose per day) Total recommended dose is 160mg/kg

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.

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.

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

There is currently nothing known about the possible total cost.

There is currently nothing known about off label use.

Indication extension	No

There is currently no futher information available.