Uveal melanoma treatment granted orphan drug status

The U.S. FDA has granted orphan drug status to a minimally invasive, outpatient treatment for uveal melanoma.

Developed by Aura Biosciences, AU-011 is an injectable nanoparticle that binds selectively to tumor cells. Infrared light from a standard ophthalmic laser causes the nanoparticle to release molecules that destroy tumor cells, leaving normal cells untouched.

Pre-clinical data presented this month at the 2015 meeting of the Association for Research in Vision and Ophthalmology demonstrated that it can treat uveal melanoma in an orthotopic xenograft model. The research team from McGill University report complete tumor eradication with 3 treatments and that treatment appeared to be “retina sparing”.

“In all of our years running this pre-clinical uveal melanoma animal model that we established more than a decade ago, this is the first time that we have seen such promising and encouraging results for the treatment of primary uveal melanoma. We are very excited about the positive impact this might have in uveal melanoma patients,” said Dr. Patrick Logan, lead investigator for the study.