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Portola Pharmaceuticals Receives FDA Orphan Drug Designation for Cerdulatinib, an Oral Syk/JAK Inhibitor for the Treatment of Peripheral T-Cell Lymphoma
“We are pleased that the
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
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 (
Portola Pharmaceuticals is a commercial-stage biopharmaceutical company focused on the discovery, development and commercialization of novel therapeutics that could significantly advance the fields of thrombosis and other hematologic diseases. The Company’s two FDA-approved medicines are Andexxa® [coagulation factor Xa (recombinant), inactivated-zhzo], the first and only antidote for patients treated with rivaroxaban and apixaban when reversal of anticoagulation is needed due to life-threatening or uncontrolled bleeding, and Bevyxxa® (betrixaban), the first and only oral, once-daily Factor Xa inhibitor for the prevention of VTE in adult patients hospitalized for an acute medical illness. The company also is advancing cerdulatinib, a Syk/JAK inhibitor for the treatment of hematologic cancers.
This announcement contains forward-looking statements, including statements regarding next steps for the development of cerdulatinib, including the potential for an accelerated approval pathway in the U.S. for certain tumor subtypes. Risks that contribute to the uncertain nature of the forward-looking statements include: failure to obtain