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

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.
Therapeutic value	No judgement yet
Total cost	9,375,000.00
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	Other hematology
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
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	June 2019
Orphan drug	Yes
Registration phase	Registration application pending
Additional remarks	Restart of the procedure 27 March 2018 - responses received to Day 120 List of Questions.

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	40 - 60 Market share is generally not included unless otherwise stated.
References	Fabrikant; EBMT activity survey 2016; Passweg 2018
Additional remarks	Hangt ervan af waarvoor het ingezet gaat worden, aantal haplo’s per centrum niet zo groot, maximaal 50-60 per jaar in Nederland daar waar het volwassenen betreft. Gaat het ingezet 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. Fabrikant: "538 allogenic HSCTs have been performed in the Netherlands in 2016, of which only 80% in adults, ie. Fabrikant: "431 patients. Circa 10% of these allogenic HSCTs were haplo-identical HSCTs in myeloid and lymfoid malignancies, ie. 43 patients. With an 2% increase in number of allogeneic HSCTs per year, this will bring the number of eligible patients to 44 for 2018."

Patient volume

40 - 60

Market share is generally not included unless otherwise stated.

References

Fabrikant; EBMT activity survey 2016; Passweg 2018

Additional remarks

Hangt ervan af waarvoor het ingezet gaat worden, aantal haplo’s per centrum niet zo groot, maximaal 50-60 per jaar in Nederland daar waar het volwassenen betreft. Gaat het ingezet 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. Fabrikant: "538 allogenic HSCTs have been performed in the Netherlands in 2016, of which only 80% in adults, ie. Fabrikant: "431 patients. Circa 10% of these allogenic HSCTs were haplo-identical HSCTs in myeloid and lymfoid malignancies, ie. 43 patients. With an 2% increase in number of allogeneic HSCTs per year, this will bring the number of eligible patients to 44 for 2018."

Expected cost per patient per year

Cost	175,000.00 - 200,000.00
References	Fabrikant

Potential total cost per year

Total cost	9,375,000.00 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.

Total cost

9,375,000.00

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.