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Donor-derived T-lymphocyte enriched leukocyte preparation depleted ex vivo of host-alloreactive T cells.

ATIR101

Extended indication Adjunctive treatment to a haploidentical, T-cell depleted haematopoietic stem cell transplantation (
Therapeutic value No judgement yet
Total cost 3,562,500.00
Registration phase Registration application pending

Product

Active substance Donor-derived T-lymphocyte enriched leukocyte preparation depleted ex vivo of host-alloreactive T cells.
Domain Oncology and Hematology
Reason of inclusion New medicine (specialité)
Main indication Stem cell transplants
Extended indication Adjunctive treatment to a haploidentical, T-cell depleted haematopoietic stem cell transplantation (HSCT) in adult patients with high-risk haematological malignancies in complete remission.
Proprietary name ATIR101
Manufacturer Kiadis
Mechanism of action Allogeneic modified cell therapy
Route of administration Intravenous
Therapeutical formulation Intravenous drip
Budgetting framework Intermural (MSZ)
Additional remarks ATIR101 is a cell-based medicinal product consisting of a donor-derived T-lymphocyte enriched leukocyte preparation depleted ex vivo of host alloreactive T-cells using photodynamic treatment (PDT), which is administered to patients who have received a haploidentical, T-cell depleted HSCT from the same donor as the donor of the cells used in ATIR101. The host alloreactive T-cells that may cause GVHD have been selectively eliminated from ATIR101. ATIR101 will provide the recipient with donor T-cell based immunity and will bridge the period of temporal immune deficiency caused by the pre-treatment until the engrafted stem cells have generated a fully functional immune system. Preservation of virus specific T-cells in ATIR101, if present in the collected donor cells, has been demonstrated in vitro for clinically relevant viruses.

Registration

Registration route Centralised (EMA)
Type of trajectory Normal trajectory
Submission date June 2017
Expected Registration September 2019
Orphan drug Yes
Registration phase Registration application pending

Therapeutic value

Current treatment options In toenemende mate wordt post allo cyclofosfamide toegepast ter preventie van GVHD in de haplo-identieke setting, dit lijkt een succesvolle en veel goedkopere benadering dan ATIR.
Therapeutic value No judgement yet
Substantiation Wordt op dit moment in gerandomiseerde studie vergeleken met cyclofosfamide maar uitkomsten zullen nog op zich laten wachten.
Duration of treatment one-off
Dosage per administration 2x10^6 cellen/kg
References NCT02500550; fabrikant, Clinical trial: CR-AIR-009.
Additional remarks ATIR101 should be infused 28 – 32 days following the receipt of a haploidentical, T-cell depleted HSCT. Lack of HLA-identical sibling or matched unrelated donors has emerged the use of haploidentical family donors. ATIR101 is the treatment option (viable T-cells) which functionally will eliminate the potentially GVHD-causing cells, decrease TRM and improve OS.

Expected patient volume per year

Patient volume

13 - 25

Market share is generally not included unless otherwise stated.
References Fabrikant; EBMT
Additional remarks In het EBMT register is te vinden dat er 21 patiënten behandeld zijn met een haploïdentieke donor in 2016 en 20 patiënten in 2017. De verwachting is dat in de periode 2019-2021 een maximum van 25 patiënten behandeld zal worden met een haploïdentieke donor op jaarbasis. Deze patiënten zullen dus mogelijk in aanmerking komen voor een behandeling. De fabrikant geeft zelf aan een marktaandeel van 50% te verwachten. Indien het ook ingezet gaat worden voor T-cel depletie in het algemeen, dus ook na bijvoorbeeld CD34+ selectie, dan in potentie meer patiënten maar dit zal veel duurder zijn dan post allo-Cy en naar verwachting heel moeilijk ingang vinden.

Expected cost per patient per year

Cost 175,000.00 - 200,000.00
References Fabrikant

Potential total cost per year

Total cost

3,562,500.00

This amount gives an indication of the total cost. It is the result of the average expected patient volume times the average cost per patient. both per year.

Off label use

Off label use No

Indication extension

Indication extension No

Other information

There is currently no futher information available.