Orphan Drug Designation Granted to Peripheral T-cell Lymphoma Treatment, Cerdulatinib

The US Food and Drug Administration (FDA) has granted an orphan drug designation to Portola Pharmaceuticals’ investigational oral tyrosine kinase and janus kinase (Syk/JAK) inhibitor, cerdulatinib, for the treatment of peripheral T cell lymphoma (PTCL).

“We are pleased that the FDA has granted cerdulatinib Orphan Drug Designation, as it recognizes its potential to provide a significant clinical benefit to a group of patients with limited treatment options,” said John Curnutte, MD, PhD, Portola’s interim co-president and head of research and development, in a recent statement.

Currently, an ongoing phase 2a clinical trial is evaluating cerdulatinib in patients with other specific subtypes of B cell and T cell Non-Hodgkin Lymphoma (NHL), such as relapsed/refractory follicular lymphoma (FL) and chronic lymphocytic lymphoma/small lymphocytic lymphoma (CLL/SLL).

In June, at both the 2018 American Society of Clinical Oncology (ASCO) Annual Meeting and at the 23rd Congress of the European Hematology Association (EHA), data were reported from the study which demonstrated the treatment’s broad clinical activity.

Specifically, cerdulatinib was found to be well tolerated and an objective response rate of 47% was recorded. A complete response rate was also observed in 7 of the 20 patients in the PTCL cohort at the time of presentation.

The important role for Syk and JAK in peripheral T-cell lymphoma (PTCL) tumor survival has been demonstrated in preclinical trials.

“We look forward to presenting additional data from the Phase 2a trial at a scientific congress early next year and to continuing discussions with the FDA regarding next steps for the development of cerdulatinib, including the potential for an accelerated approval pathway,” Dr. Curnutte added.

PTCLs are a group of rare, aggressive types of non-Hodgkin lymphomas (NHL) that develop in T cells and natural killer cells, according to the Leukemia & Lymphoma Society. About 10% to 15% of all patients with NHL have a T cell lymphoma subtype.