Timrepigene emparvovec

Extended indication	Maat013 monotherapy for salvage treatment of gastrointestinal Acute graft versus host disease in adults and elderly who are refractory to both steroids, the standard of care used as first-line treatment, and to ruxolitinib used as a second-line treatment
Therapeutic value	No estimate possible yet
Registration phase	Clinical trials

Product

Active substance	MaaT013
Domain	Hematology
Reason of inclusion	New medicine (specialité)
Main indication	Graft versus Host
Extended indication	Maat013 monotherapy for salvage treatment of gastrointestinal Acute graft versus host disease in adults and elderly who are refractory to both steroids, the standard of care used as first-line treatment, and to ruxolitinib used as a second-line treatment
Manufacturer	MaaTPharma
Route of administration	Rectal
Budgetting framework	Intermural (MSZ)
Additional remarks	Suspensie voor rectaal gebruik.

Registration route	Centralised (EMA)
Type of trajectory	Normal trajectory
ATMP	No
Submission date	February 2025
Expected Registration	March 2026
Orphan drug	Yes
Registration phase	Clinical trials
Additional remarks	Indieningsdatum en verwachte registratie op basis van IHSI-inschatting.

Therapeutic value	No estimate possible yet
References	NCT04769895 (ARES)

There is currently nothing known about the expected patient volume.

There is currently nothing known about the expected cost.

There is currently nothing known about the possible total cost.

There is currently nothing known about off label use.

There is currently nothing known about indication extensions.

There is currently no futher information available.