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news.US-based biotechnology firm Bio-Path Holdings have secured orphan drug designation from the US Food and Drug Administration (FDA) for its lead compound, Liposomal Grb-2, to treat acute myeloid leukemia (AML).
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Currently, Liposomal Grb-2 is in a safety segment of a Phase II trial in combination with Ara-C for the treatment of AML. It is also being evaluated for chronic myelogenous leukemia.
The company said that pre-clinical development is ongoing for triple negative and inflammatory breast cancers.
With the FDA orphan drug status, the company will have seven years of exclusivity after receiving formal marketing approval, as well as additional development incentives.
Bio-Path president and chief executive officer Peter Nielsen said: "This designation from the FDA demonstrates the unmet need for an effective therapy for patients suffering from AML.
"It also marks a key regulatory milestone for Bio-Path and will be valuable as we continue to progress Liposomal Grb-2 through clinical trials and toward potential commercialization."
AML is a fast-growing cancer of the blood and bone marrow in which the bone marrow makes many cancerous cells called leukemic blasts.
Normal blasts develop into white blood cells that fight infection, while in AML, the leukemic blasts do not develop properly and cannot fight infections.