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Roswell Park spinoff drug under study gets FDA orphan status

The Food and Drug Administration has awarded orphan drug status to an immunotherapy vaccine, SurVaxM, to treat the type of brain cancer known as a glioblastoma.

SurVaxM was developed by MimiVax LLC, a Roswell Park Cancer Institute spinoff company formed by Dr. Robert Fenstermaker and his Roswell Park colleague, Michael Ciesielski. The potential therapy targets a molecule, survivin, that is present in brain tumors and other difficult-to-treat, often-fatal tumors.

Survivin is a substance naturally found in the body, so the immune system doesn’t respond to its presence. The vaccine is engineered to stimulate an immune response to kill cancer cells that contain survivin and control tumor growth.

Orphan status is aimed at encouraging companies to develop new drugs for rare diseases. Its benefits include seven years of marketing exclusivity after approval of a product for sale.

A phase II study of SurVaxM, in addition to standard treatment for newly diagnosed glioblastoma, is under way to see if the therapy works at five medical institutions, including Roswell Park. SurVaxM is also being studied in another clinical trial as part of combination therapy for multiple myeloma.

Glioblastomas in adults account for 35 percent to 40 percent of malignant brain tumors, with about 14,000 cases diagnosed each year in the United States, according to the National Cancer Institute.

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