Allos Therapeutics' FOLOTYN™ First And Only FDA-Approved Therapy For Relapsed Or Refractory Peripheral T-cell Lymphoma

Main Category: Lymphoma / Leukemia / Myeloma
Also Included In: Regulatory Affairs / Drug Approvals;  Blood / Hematology
Article Date: 28 Sep 2009 - 4:00 PDT

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Allos Therapeutics, Inc. (Nasdaq:ALTH) announced that last night the U.S. Food and Drug Administration (FDA) granted accelerated approval for FOLOTYNTM (pralatrexate injection) for use as a single agent for the treatment of patients with relapsed or refractory peripheral T-cell lymphoma (PTCL). FOLOTYN is the first and only drug approved by the FDA for this indication and represents a new treatment option for patients with relapsed or refractory PTCL. This indication is based on overall response rate. Clinical benefit such as improvement in progression free survival or overall survival has not been demonstrated. Allos expects to make FOLOTYN available to patients in the U.S. in October.

"Individuals with peripheral T-cell lymphoma have a very poor prognosis and almost always relapse or become refractory to initial therapy.1 As a result, there is an urgent need for new therapies to treat patients with this challenging disease. FOLOTYN has demonstrated its efficacy and safety in the PROPEL clinical trial, and I believe it will be a welcome addition for physicians who treat patients with relapsed or refractory PTCL," stated Owen A. O'Connor, MD, PhD, principal investigator in the PROPEL study of FOLOTYN; deputy director for Clinical Research and Cancer Treatment, NYU Cancer Institute; chief, Division of Hematologic Malignancies and Medical Oncology; professor of Medicine and Pharmacology at the NYU Langone Medical Center.

PTCL comprises a biologically diverse group of aggressive blood cancers that has a poor prognosis.2 The Company's New Drug Application (NDA) for FOLOTYN was based on data from the PROPEL trial. The Company believes PROPEL is the largest prospective, multicenter, international trial ever conducted in patients with relapsed or refractory PTCL.

"We are enthusiastic about providing this new therapy to patients with relapsed or refractory PTCL," said Paul L. Berns, president and chief executive officer at Allos Therapeutics, Inc. "The approval of FOLOTYN is a transformative event for Allos representing our first U.S. indication. We thank the many patients and clinical investigators who participated in the PROPEL study. Moving forward, we plan to continue advancing the FOLOTYN clinical development program."

"Aggressive peripheral T-cell lymphomas have been a largely ignored group of diseases," said James O. Armitage, MD, The Joe Shapiro Professor of Medicine, Department of Internal Medicine, University of Nebraska Medical Center. "It is exciting to have the first FDA-approved therapy for relapsed or refractory peripheral T-cell lymphoma."

Allos is dedicated to patient access and has established a patient assistance program named ASAP (Allos Support for Assisting Patients) to provide reimbursement support. Commencing in October, more information regarding ASAP will be available by calling the Hotline at 1-877-ASAP102 (272-7102), Monday to Friday, 8 a.m. to 7 p.m. Central Time or by visiting http://www.getASAPinfo.com.

"The approval of FOLOTYN brings a new treatment option to patients afflicted with peripheral T-cell lymphoma," said Peter L. Saltonstall, president and chief executive officer of the National Organization for Rare Disorders (NORD). "We at NORD are excited about this approval and will continue our efforts to focus national attention on rare diseases and on the fact that most rare diseases have no FDA-approved treatment at this time."

In connection with the accelerated approval, Allos has agreed to undertake additional clinical studies to further verify and describe the clinical benefit of FOLOTYN in patients with T-cell lymphoma.

FOLOTYN was discovered by Sloan-Kettering Institute for Cancer Research, SRI International and Southern Research Institute and developed by Allos Therapeutics.

About PROPEL

The FOLOTYN approval was based on the results from PROPEL, an open-label, single-arm, multi-center, international clinical trial that enrolled 115 patients with relapsed or refractory PTCL, 109 of whom were considered evaluable for efficacy according to the trial protocol. Patients were considered evaluable if they received at least one dose of FOLOTYN, their diagnosis of PTCL was confirmed by independent pathology review, and they had relapsed or refractory disease after at least one prior treatment. Patients were treated with FOLOTYN at 30 mg/m2 once weekly by IV push over 3-5 minutes for 6 weeks in 7-week cycles until disease progression or unacceptable toxicity. In addition, patients received 1mg 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 (complete response, complete response unconfirmed and partial response) as assessed by International Workshop Criteria (IWC). The key secondary efficacy endpoint was duration of response. Response assessments were scheduled at the end of cycle 1 and then every other cycle (every 14 weeks). Duration of response was measured from the first day of documented response to disease progression or death. Response and disease progression were evaluated by independent central review using the IWC.

The results of the trial demonstrated that 29 of 109 evaluable patients, or 27%, responded to FOLOTYN. The median duration of response was 287 days, or 9.4 months (range 1-503 days). Thirteen of 109 evaluable patients had a duration of response ≥ 14 weeks (range 98-503 days). 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. See below for Important Safety Information.

The median number of prior systemic therapies was 3 (range 1-12). Approximately one-fourth of patients (24%, n = 27) did not have evidence of response to any previous therapy. Approximately two-thirds of patients (63%, n = 70) did not have evidence of response to their most recent prior therapy before entering the trial. The initial response assessment was scheduled at the end of cycle 1. Of the responders, 66% responded within cycle 1. The median time to first response was 45 days (range 37-349 days).

Source
Allos Therapeutics, Inc.