Spectrum Pharmaceuticals, with a primary focus in Hematology and Oncology, announced today that the U.S. Food and Drug Administration (FDA) has granted Accelerated Approval of Beleodaq™ for the treatment of patients with relapsed or refractory peripheral T-cell lymphoma (PTCL). This indication is approved under accelerated approval based on Tumor Response Rate and Duration of Response . An improvement in survival or disease-related symptoms has not been established. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trial.

Beleodaq was approved by the FDA on July 3 rd, nearly 5 weeks before the PDUFA date (August 9 th ). This indication was approved based on data from the multi-center, single-arm BELIEF trial in 120 evaluable patients, refractory to or who had failed at least one prior systemic therapy. In this trial, Beleodaq was associated with hematologic toxicity, infections, hepatotoxicity, tumor lysis syndrome, gastrointestinal toxicity, and embryo-fetal toxicity.

PTCL comprises a group of rare and aggressive non-Hodgkin’s Lymphomas (NHL) that develop from mature T-cells and accounts for approximately 10 to 15% of all NHL cases in the United States. These patients generally have a poor prognosis with a low response rate (25-27%) to available treatment options, and commonly experience repeated treatment failures until drug resistance or death. Therefore, there has been an important unmet medical need for these patients with PTCL for additional new treatment options that are specifically effective for this disease.

“This FDA approval enables us to help address this unmet medical need, and provide a new treatment option for patients with this difficult-to-treat and ultimately fatal disease,” said Rajesh C. Shrotriya, MD, Chairman and Chief Executive Officer of Spectrum Pharmaceuticals. “First with Folotyn ® (pralatrexate injection) and now with Beleodaq, we are very proud to be able to offer patients and clinicians two approved treatment options for R/R PTCL, and be a leader in the treatment of T-cell lymphomas. We will be able to effectively leverage our existing Hematology clinical and sales infrastructure to expedite the launch of Beleodaq. Now with a total of five approved Hematology/Oncology drugs and a strong and maturing development pipeline, Spectrum is well positioned for continued future growth.”

“Peripheral T-cell lymphoma (PTCL) is a poor prognosis subtype of non-Hodgkin's lymphoma with no accepted standard of care,” said Owen A. O'Connor, MD, PhD, Director of Lymphoid Malignancies, Professor of Medicine and Experimental Therapeutics at Columbia Medical Center, New York Presbyterian Medical Center, one of the lead investigators in the BELIEF study. “Relapse is common after initial treatment, and there are limited options for patients in 2 nd line and beyond. Histone deacetylase inhibitors have emerged as one promising class of drugs for patients faced with this disease. One interesting observation in the study was the tolerability of Beleodaq in these heavily treated patients. Beleodaq was associated with myelosuppression with an overall rate of anemia of 32%, thrombocytopenia of 16.3% and neutropenia of 9.3% and Grade 3/4 adverse reactions were reported in 10.9%, 7.0% and 6.2% of patients, respectively. The associated severity of hematologic toxicities may prove to be useful in previously treated patients who have poor bone marrow reserve.”

“Interestingly, Beleodaq was shown to have an Overall Response Rate of 25.8% with a high response rate (45.5%) in patients with Angioimmunoblastic T-cell Lymphoma, one of the common PTCL subtypes. In addition, 17% of the patients enrolled in this trial had low Baseline platelet counts (<100,000/mm 3 ) and tolerated therapy with some (15%) attaining partial and complete responses. I believe Beleodaq will be a valuable new option for physicians who treat patients with relapsed or refractory PTCL. This safety profile makes it a potential candidate for the development of new combination treatment paradigms for patients with PTCL,” added Dr. O'Connor.

A review of data from a planned confirmatory Phase III trial of Beleodaq in combination with CHOP (cyclophosphamide, vincristine, doxorubicin, prednisone), to characterize the efficacy and safety of the Beleodaq combination versus CHOP alone, is required by FDA to convert this Accelerated Approval to a Full Approval.