Drug has been shown to improve overall response but not progression-free or overall survival.

FDA granted Allos Therapeutics accelerated approval for Folotyn as a single agent for the treatment of relapsed or refractory peripheral T-cell lymphoma (PTCL). The decision was based on data showing a positive overall response rate. Improvement in progression-free survival and overall survival, however, have not been demonstrated.

Allos expects to make Folotyn available to patients in the U.S. in October. In connection with the accelerated approval, Allos will undertake additional clinical studies to further verify and describe the clinical benefit of Folotyn in T-cell lymphoma.

Folotyn was discovered by Sloan-Kettering Institute for Cancer Research, SRI International, and Southern Research Institute and developed by Allos Therapeutics. The company is also studying the compound in B-cell non-Hodgkin’s lymphoma (Phase II), cutaneous T-cell lymphoma (Phase I), and bladder cancer (Phase II). Additionally, Folotyn plus gemcitabine is being investigated in non-Hodgkin’s lymphoma (Phase I), and Folotyn plus erlotinib in non-small-cell lung cancer (Phase II).

The FDA go-ahead marks the first medication sanctioned for peripheral T-cell lymphoma, according to the company. The decision was based on an open-label, single-arm, multicenter, international trial that enrolled 115 patients with 109 of them considered evaluable.

Patients were treated with Folotyn at 30 mg/m2 once weekly by IV over three to five minutes for six weeks in seven-week cycles until disease progression or unacceptable toxicity. In addition, patients received 1 mg of vitamin B12 intramuscularly every 8–10 weeks and 1.0–1.25 mg of folic acid orally on a daily basis. The primary efficacy endpoint was overall response rate, which includes complete response, complete response unconfirmed, and partial response. The key secondary efficacy endpoint was duration of response.

The trial demonstrated that 29 patients, or 27%, responded to Folotyn. The median duration of response was 287 days, or 9.4 months. Of the 109 evaluable patients, 13 had a duration of response ≥ 14 weeks. The most common grade 3/4 adverse events were thrombocytopenia, which was observed in 33% of patients, mucositis in 21% of patients, neutropenia in 20% of patients, and anemia in 17% of patients.

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