Extended indication

Relapsed and Refractory Synovial Sarcoma Expressing New York Esophageal Antigen-1 (NY-ESO-1) and/or

Therapeutic value

No estimate possible yet

Registration phase

Clinical trials

Product

Active substance

NY-ESO-1 autologous engineered TCR-T cells

Domain

Oncology and Hematology

Reason of inclusion

New medicine (specialité)

Main indication

Oncology other

Extended indication

Relapsed and Refractory Synovial Sarcoma Expressing New York Esophageal Antigen-1 (NY-ESO-1) and/or LAGE-1a.

Manufacturer

GSK

Mechanism of action

Autologous modified cell therapy

Route of administration

Intravenous

Therapeutical formulation

Intravenous drip

Budgetting framework

Intermural (MSZ)

Additional comments
NY-ESO-1 autologous engineered TCR-T cells (engineered TCR).

Registration

Registration route

Centralised (EMA)

Type of trajectory

Accelerated assessment

Particularity

New medicine with Priority Medicines (PRIME)

ATMP

Yes

Submission date

2022

Expected Registration

2023

Orphan drug

Yes

Registration phase

Clinical trials

Therapeutic value

Current treatment options

Bestraling en chemotherapie.

Therapeutic value

No estimate possible yet

Duration of treatment

one-off

Dosage per administration

1 tot 6 x10^9 cellen

References
NCT03697824
Additional comments
De fase 2 studie NCT03697824 is teruggetrokken en wordt vervangen door een studie naar monotherapie.

Expected patient volume per year

Patient volume

5

Market share is generally not included unless otherwise stated.

References
NKR;
https://mens-en-gezondheid.infonu.nl/aandoeningen/187822-synoviaal-sarcoom-kanker-van-weefsel-rond-gewrichten.html#behandeling;
Lai et al. Oncoimmunology. 2012 Nov 1; 1(8): 1409–1410.
Additional comments
In 2016 waren er 34 wekedelensarcomen van de extremiteiten gediagnosticeerd, waar het synoviaal carcinoom er een van is. De incidentie van dit synoviaal carcinoom wordt geschat op 1 in de 3 miljoen, ongeveer 6 per jaar in Nederland, Expressie van NY-ESO-1 komt voor in ongeveer 80% van de gevallen van synoviaal sarcoom (5 patiënten).

Expected cost per patient per year

Additional comments
Gezien de PRIME-status zal deze behandeling waarschijnlijk hoge kosten met zich meebrengen.

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

Yes

Indication extensions

Gastrointestinal cancer, Malignant melanoma, Multiple myeloma, Non-small cell lung cancer, Ovarian cancer, Liposarcoma.

References
AdisInsight

Other information

There is currently no futher information available.