AbbVie and Janssen Biotech receive fourth FDA Breakthrough Therapy Designation for imbruvica

AbbVie and Janssen Biotech have received the fourth Breakthrough Therapy Designation (BTD) from the US Food and Drug Administration (FDA) for ibrutinib (imbruvica), as an effective treatment of chronic graft-versus-host-disease (cGVHD).

Imbruvica is a first-in-class, oral, once-daily therapy that inhibits a protein called Bruton's tyrosine kinase (BTK), which is a major signalling molecule in the B-cell receptor signalling complex, playing an important part in the survival and spread of malignant B cells.

The drug helps block these signals that encourage malignant B cells to multiply and spread uncontrollably.

cGVHD is a serious and life-threatening condition whereby transplanted cells from the donor attack the patient's body after the patient undergoes allogeneic stem cell or bone marrow transplantation.

The therapy is used after the failure of one or more lines of systemic therapy, and has also been granted the Orphan Drug Designation (ODD) for the cGVHD condition by the FDA.

Ibrutinib (imbruvica) has been developed, as well as commercialised, by a collaboration of Pharmacyclics, a subsidiary of Chicago-based AbbVie, and Janssen.

There are currently no particular therapies approved for this condition, while most of the patients afflicted with the condition are prescribed glucocorticoids, a systemic steroid treatment that acts upon cells throughout the body.

Pharmacyclics oncology head Dr Danelle James said: "This fourth Breakthrough Therapy Designation from the FDA shows the promise of imbruvica and its unique mechanism of action as a potential therapy beyond blood cancers, including chronic graft-versus-host-disease, a severe inflammatory condition with currently no approved therapies specifically for these patients.

"We are committed to continuing to evaluate the potential benefit ibrutinib may offer in treating blood cancers, solid tumours and other health conditions with unmet medical needs."

In February 2013, the FDA granted the first and second BTD to imbruvica to treat patients affected with relapsed or refractory mantle cell lymphoma (MCL) and for the treatment of patients with Waldenström's macroglobulinemia (WM).

In April 2013, imbruvica received its third BTD for chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL) with a deletion of the short arm of chromosome 17 (del 17p).

The FDA also assigned imbruvica ODD for all three indications.