The US Food and Drug Administration (FDA) has granted Orphan Drug Designation to BN-Brachyury, a novel vaccine, for the treatment of chordoma, a rare bone cancer that shares characteristics with sarcomas.1

Recent research suggests that the brachyury protein may be a driver of chordoma, which can be difficult to resect because these tumors occur at the sacrum, mobile spine, or clivus. The BN-Brachyury vaccine targets this protein, and is being evaluated in a phase 1 trial ( Identifier: NCT03349983) for assessment of the treatment’s safety and tolerability.

Researchers plan to initiate a phase 2 trial of the vaccine, which will enroll up to 25 patients with metastatic chordoma. BN-Brachyury will be evaluated in combination with radiotherapy.

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While chordoma is diagnosed in only about 300 patients in the United States each year, the disease carries a poor prognosis. Cancers driven by brachyury are, moreover, more likely to be resistant to standard therapies.

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The FDA’s Orphan Drug Designation is reserved for treatments likely to be safe and effective in diseases that affect fewer than 200,000 patients per year in the US. BN-Brachyury was developed by Bavarian Nordic in collaboration with the National Cancer Institute (NCI).

More information about clinical trials of BN-Brachyury can be found on the NCI’s website.


  1. Bavarian Nordic receives FDA Orphan Drug Designation for BN-Brachyury for the treatment of chordoma [news release]. Copenhagen, Denmark: Bavarian Nordic; May 2, 2018. Accessed May 2, 2018.