Portola Pharmaceuticals (Nasdaq: PTLA) today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to cerdulatinib, an investigational, oral Syk/JAK inhibitor 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, M.D., Ph.D., Portola’s interim co-president and head of research and development. “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.”
The FDA's Office of Orphan Products Development grants orphan status to support development of medicines for the treatment of rare diseases that affect fewer than 200,000 people in the United States. Orphan Drug Designation may provide certain benefits, including a seven-year period of market exclusivity if the drug is approved, tax credits for qualified clinical trials and an exemption from FDA market application fees.
PTCL is a group of rare and often fast-growing lymphomas classified as a subtype of non-Hodgkin’s lymphoma. There are approximately 6,000 to 10,000 cases of PTCL annually in the United States. The three most common types of PTCL are anaplastic large cell lymphoma (ALCL), angioimmunoblastic T-cell lymphoma (AITL), and PTCL not otherwise specified (PTCL-NOS), but many other rarer types exist. PTCL is most often treated with a combination of chemotherapies and the options for patients that fail front-line therapy are limited.
Cerdulatinib is an investigational oral, dual spleen tyrosine kinase (Syk) and janus kinase (JAK) inhibitor that uniquely inhibits two key cell signaling pathways implicated in certain hematologic malignancies and autoimmune diseases. There is a strong rationale for inhibiting both Syk (B-cell receptor pathway) and JAK (cytokine receptors) in B-cell malignancies where both targets have been shown to promote cancer cell growth and survival. In addition, pre-clinical data suggest an important role for Syk and JAK in peripheral T-cell lymphoma (PTCL) tumor survival.
In addition to PTCL, cerdulatinib is being evaluated in an ongoing Phase 2a study among patients with other specific subtypes of B-cell and T-cell Non-Hodgkin Lymphoma (NHL), including relapsed/refractory follicular lymphoma (FL) and chronic lymphocytic lymphoma/small lymphocytic lymphoma (CLL/SLL). The Company reported new data from this study 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). Cerdulatinib demonstrated broad clinical activity including an objective response rate of 47 percent in all patients, and was generally well tolerated. Additionally, seven of the 20 patients in the PTCL cohort achieved a complete response at the time of presentation.