2017 is Proving a Big Year for Orphan Drug Designation: Here’s Another One!

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In recent news in the oncology community, the FDA has approved orphan drug designation to a new drug, SurVaxM, for glioblastoma.

Glioblastoma is a rare type of brain cancer that forms from star-shaped astrocyte cells.

There are usually highly malignant and often regrow after treatment. For these reasons, a better treatment option, especially for resistant and refractory glioblastoma, is desperately needed.

That’s why this new designation for SurVaxM is so important. There are a couple of aspects to this therapy option, in particular, that help distinguish it from the current treatments out there and suggest that it could be a much more effective option.

In broad terms, SurVaxM is an immunotherapy that has been engineered to control the growth of tumors and their recurrence by stimulating the immune system in multiple different ways, namely, targeting a protein called survivin. Survivin actually allows cancer cells to resist and fight against conventional treatments, and it is found in multiple different kinds of cancer.

Thus, while the Orphan Drug designation is approved only for glioblastoma right now, SurVaxM could have a much broader application in the future.

But so far, the drug has been evaluated in patients with newly-diagnosed glioblastoma and multiple myeloma. Scientists are hoping to conduct later stage trials soon, especially given this new designation, which only fuels the momentum of this research.

To read more about the FDA’s ruling on SurVaxM, read this article here.


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