The U.S. Food and Drug Administration (FDA) has granted orphan drug status to Portola Pharmaceuticals‘ investigative therapy cerdulatinib to treat patients with peripheral T-cell lymphoma (PTCL), the company announced.
Orphan drug designation aims to encourage therapies for rare and serious diseases, through benefits such as seven years of market exclusivity and exemption from FDA application fees.
“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,” John Curnutte, MD, PhD, Portola’s interim co-president and head of research and development, said in a press release.
Cerdulatinib is an oral therapy that works by inhibiting the activity of two different enzymes — the spleen tyrosine kinase (SYK) and the janus kinase (JAK) — that promote tumor growth and survival in certain blood malignancies and autoimmune diseases.
The therapy is being tested in a Phase 1/2a trial (NCT01994382), which is testing cerdulatinib in multiple lymphoma types, including PTCL, cutaneous T-cell lymphoma, chronic lymphocytic lymphoma/small lymphocytic lymphoma, aggressive non-Hodgkin’s lymphoma, and B-cell non-Hodgkin’s lymphoma. All participants have failed to respond to several prior lines of therapy.
In the first part of the trial, completed in 2016, researchers confirmed that cerdulatinib was well-tolerated and had anti-tumor activity in several blood cancer types.
The second part of the trial focused on confirming the safety and effectiveness of cerdulatinib, dosed at 30 mg twice a day, by patients who had received at least three prior therapies.
Interim results have shown that 47% of patients responded to the treatment. For the 20 patients with PTCL, 43% experienced a reduction in tumor burden, including seven who had a complete tumor clearance.
Again, the medicine was well-tolerated, with the most common side effects being increased levels of an enzyme called lipase, low counts in a group of immune cells called neutrophils, and pneumonia.
However, five deaths due to sepsis or septic shock (three of which were concomitant with pneumonia) were attributed to cerdulatinib. These occurred primarily in patients with CLL/SLL.
“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,” Curnutte said.