FDA Grants Orphan Drug Designation to BN-Brachyury for Chordoma
Researchers plan to initiate a phase 2 trial of the vaccine, which will enroll up to 25 patients with metastatic chordoma.
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 (ClinicalTrials.gov 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.
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.
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.
- Bavarian Nordic receives FDA Orphan Drug Designation for BN-Brachyury for the treatment of chordoma [news release]. Copenhagen, Denmark: Bavarian Nordic; May 2, 2018. http://www.bavarian-nordic.com/investor/news/news.aspx?news=5433. Accessed May 2, 2018.