FDA advisory committee unanimously recommends approval of Amgen's NEUPOGEN ® for peripheral blood progenitor cell support; recent data showed 30% cost savings over BMT

Business Wire, Nov 13, 1995

THOUSAND OAKS, Calif.--(BUSINESS WIRE)--Nov. 13, 1995--Amgen (NASDAQ:AMGN) Monday announced that the Biological Response Modifier's Advisory Committee (BRMAC) to the Food and Drug Administration voted unanimously (15-0) to recommend expanding the approved use of the company's recombinant human granulocyte colony-stimulating factor NEUPOGEN (R) (Filgrastim rG-CSF) to include use in peripheral blood progenitor cell (PBPC) transplantation.

NEUPOGEN-mobilized PBPC transplantation is an advance in stem cell transplant that is replacing bone marrow transplant (BMT) procedures, because it offers a faster patient recovery, and according to a recent study, does so at lower cost.

The recommendation, if approved by the FDA, would expand use of NEUPOGEN in mobilizing PBPCs in order to provide more rapid recovery of the blood's white cells (neutrophils) and clotting cells (platelets) than BMT following some forms of intensive cancer chemotherapy that suppress the body's ability to form certain blood cells. In data presented to the Advisory Committee, NEUPOGEN- mobilized PBPCs, followed by post-transplant administration of NEUPOGEN, was shown to reduce both the time of hospitalization and the number of platelet transfusions.

High doses of chemotherapy are often needed to treat some forms of cancer but often destroy the ability of the bone marrow to manufacture neutrophils which are needed to prevent and combat potentially severe infections, and platelets which prevent bleeding.

Physicians increasingly elect to transplant NEUPOGEN generated PBPCs, sometimes called "parent" cells because they give rise to a variety of later cells, including neutrophils, red cells and platelets, speed the production of these later cells following these marrow-compromising or myeloablative forms of chemotherapy, and protect patients against potentially severe infections and bleeding complications.

Previously, cell transplants were performed by surgically removing progenitor cells from the pelvis of either the patient or a donor and then transplanting the blood cells to the patient following myeloablative chemotherapy. However, these bone marrow transplants are highly invasive, uncomfortable, require prolonged hospitalization and result in charges of $150,000 or more. With the PBPC transplant procedure, these cells are removed from the blood instead of from the marrow, a less invasive procedure done on an out-patient basis.

Under normal conditions, only small numbers of these blood- forming PBPCs are present in the blood. However, NEUPOGEN has been shown in clinical studies to increase the release of cells from the bone marrow into the circulation. They can then be "harvested" from the blood and stored, using a relatively simple procedure that does not require anesthesia.

After patients have undergone high-dose chemotherapy to kill tumor cells, they receive infusions of the stored PBPCs with NEUPOGEN, which speed recovery of the depleted infection-fighting neutrophils and platelets. As a result of PBPC infusion, there is less need for platelet transfusions. Moreover, for many patients, faster blood cell recovery results in fewer days in the hospital following re-infusion.

"Approval of this indication would provide physicians with an important advance in their cancer treatment arsenal because the use of Filgrastim-mobilized PBPC would enable oncologists to safely deliver higher doses of chemotherapy," said George Morstyn, M.D., Vice President, Clinical Development and Chief Medical Officer.

"The use of NEUPOGEN in this setting improves the ability of patients to sustain higher PBPC-supported doses of chemotherapy. These are significant clinical advances for the physician and provide quality of life benefits for patients," Dr. Morstyn said.

The procedure also has been shown to provide a cost advantage over BMT. Results of a 1994 randomized, placebo-controlled study comparing BMT with Filgrastim and PBPC with Filgrastim showed that PBPC patients had significantly shorter hospitalization (17 vs. 23 days), time to neutrophil recovery (11 vs. 14 days) time to platelet recovery (16 vs. 23 days) and fewer platelet transfusion days (six vs. 10.5 days).

In this trial, resource utilization data was collected prospectively in order to evaluate the economic implications of PBPC. In a study presented at last May's annual meeting of the American Society of Clinical Oncology (ASCO), it was shown that the mean cost and charges associated with the PBPC procedure are 30 percent lower than the mean costs and charges of the BMT procedure.

In January, the European Committee on Proprietary Medicinal Products (CPMP) recommended by a 12-0 vote approval of NEUPOGEN for PBPC transplantation in the European Union. Most of the individual countries have since formally approved this use, including the United Kingdom, Ireland, Spain, Luxembourg, Austria, the Netherlands, Sweden, Switzerland, France, Germany, Belgium and Denmark.

NEUPOGEN is already marketed in the United States as an adjunct to chemotherapy. It is used to decrease the incidence of infection, as manifested by febrile neutropenia, in patients with non-myeloid malignancies receiving myelosuppressive anti-cancer drugs associated with a significant incidence of severe neutropenia with fever and to reduce the duration of neutropenia following allogeneic and autologous bone marrow transplantation.