Janssen's Imbruvica has picked up a fourth breakthrough designation as well as Orphan status from US regulators as a treatment for chronic graft-versus-host-disease (cGVHD).
The drug is being developed as monotherapy for patients who have failure to respond to one or more lines of systemic therapy, marking its first step outside of the field of haematological malignancies.
GVHD is a life-threatening condition in which the body is attacked by donor immune cells after a patient undergoes an allogeneic stem cell or bone marrow transplant, but currently there are no approved options specifically for patients who have failed first-line corticosteroid therapy.
Imbruvica (ibrutinib) works by blocking a specific protein called Bruton's tyrosine kinase (BTK), one of the key kinase enzymes also believed to be involved in the cell signalling associated with cGVHD; its breakthrough badge in this indication was granted on the back of data from a Phase Ib/II study, which showed early clinical activity in the reduction of cGVHD, the firm noted.
Imbruvica also carries breakthrough designations for the treatment of patients with relapsed or refractory mantle cell lymphoma, Waldenström's macroglobulinemia and chronic lymphocytic leukaemia or small lymphocytic lymphoma (SLL) with a del 17p mutation.