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Title: FDA Advisory Committee Recommends Accelerated Approval of GlaxoSmithKline's Arranon (nelarabine) Injection

"FDA Advisory Committee Recommends Accelerated Approval of GlaxoSmithKline's Arranon (nelarabine) Injection"

Orphan Drug Under Review for Children and Adults with T-cell Acute Lymphoblastic Leukemia (T-ALL) and T-cell Lymphoblastic Lymphoma (T-LBL) BETHESDA, MD, and PHILADELPHIA, PA -- September 15, 2005 -- GlaxoSmithKline announced that the Oncologic Drugs Advisory Committee (ODAC) to the United States Food and Drug Administration (FDA) recommended the accelerated approval of Arranon(R) (nelarabine) Injection, a chemotherapy agent. GSK is seeking approval in the United States of Arranon for the treatment of T-cell acute lymphoblastic leukemia (T-ALL) and T-cell lymphoblastic lymphoma (T-LBL) in pediatric and adult patients whose disease has not responded to or has relapsed following treatment with at least two chemotherapy regimens. By a vote of 11 to 1, the advisory committee recommended that accelerated approval be granted in the pediatric population. The committee was unanimous in recommending accelerated approval for the adult population. "We are extremely pleased that the advisory committee has recommended the approval of Arranon," said Paolo Paoletti, M.D., Senior Vice President of the Oncology Medicine Development Center, at GSK. "Today's decision brings us one step closer to fulfilling our company's commitment to providing a new treatment option for these patients suffering from a rare and deadly disease." Discovered by GSK, Arranon is a water-soluble prodrug of ara-G with T-cell selectivity. Arranon has been studied in collaboration with the National Cancer Institute (NCI) of the National Institutes of Health (NIH), the Children's Oncology Group (COG) and the Cancer and Leukemia Group B (CALGB), in conjunction with the Southwest Oncology Group (SWOG). Throughout the development of the compound, more than 980 patients have been treated with Arranon. The clinical studies were sponsored by the Cancer Therapy Evaluation Program of the NCI under a Clinical Trials Agreement between GSK and the NCI. In December 2003, the FDA granted Arranon Fast Track designation, conferred on products in development that demonstrate a potential to address an unmet medical need for a serious or life-threatening condition. The FDA later granted Arranon Orphan Drug Status, a designation given to products under development for a rare disease or condition (i.e. affecting fewer than 200,000 people per year in the United States). It is estimated that 1,600 adults and children are diagnosed in the United States every year with these rare cancers, T-ALL and T-LBL(i). "There simply aren't many options currently available for these treatment- resistant cancers, especially in children, therefore the ODAC's recommendation today is very encouraging," said Richard Larson, Professor of Medicine, Director Hematologic Malignancies Program, CALGB Leukemia Committee Chair, The University of Chicago, Pritzker School of Medicine. "If approved by the FDA, Arranon will be a welcome addition in our fight against cancer." The ODAC reviewed data from two, multi-center pivotal Phase II clinical trials evaluating a total of 39 adults and 151 children with T-ALL or T-LBL. The trials were conducted by cooperative groups under the sponsorship of the NCI. The efficacy data to support the proposed indication focuses on 28 adults and 39 children that had multiple relapses following, or were refractory to, at least two prior induction regimens. Key efficacy results showed that 21% of the adults and 23% of the children achieved a complete response (CR) or a complete response without full hematological recovery (CR*) with single agent Arranon. A majority of those were CRs (18% in adults and 13% in pediatric patients). For this treatment setting, remissions were considered durable and generally long enough to allow for stem cell transplant procedure, often the intent following successful induction of remission. Following Arranon, patients had a median overall survival of 21 weeks for adults and 13 weeks for children and 1-year survival rates of 29% in adults and 14% in children. Hematologic toxicity was the most common NCI Common Toxicity Criteria Grade 3 (moderate) or 4 (severe) adverse event in the studies. Consistent with other cytotoxic agents, Arranon is associated with neurological events, some considered severe. Dosing of Arranon was discontinued when neurologic events of Grade 2 or greater persisted. GSK believes that the safety profile is acceptable at the proposed doses for this heavily pretreated patient population. About Acute Lymphoblastic Leukemia (ALL) and Lymphoblastic Lymphoma (LBL) Acute lymphoblastic leukemia (ALL) is a cancer of the white blood cells, the cells in the body that normally fight infections. It is an aggressive disease that is more common in children and progresses rapidly in the absence of effective therapy. ALL is the most common cancer in children, representing 23% of cancer diagnoses among children younger than 15 years of age(ii). T-ALL patients tend to have a poor prognosis and represent a subset of the ALL population. Lymphoblastic lymphoma (LBL) is a type of non-Hodgkin's lymphoma (NHL), a cancer of the lymphatic system, which occurs more often in children than adults. T-LBL patients represent a subset of this population. SOURCE: GlaxoSmithKline

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