The US Food and Drug Administration (FDA) granted Orphan Drug Designation to Aura Biosciences' drug AU-011 for the treatment of uveal melanoma. The FDA’s Orphan Drug Designation programme provides orphan status to drugs and biologics, which demonstrate promise for the diagnosis and/or treatment of rare diseases or conditions in the US.
Uveal melanoma, also referred to as ocular melanoma (OM), Uveal melanoma is the most common primary intraocular malignancy in adults. The primary tumour is diagnosed when it is still in the eye but has no targeted therapies available and is usually treated with an invasive radioactive plaque placed against the exterior of the eye near the tumour. This treatment requires multiple surgeries and can lead to cataracts, retinopathy and loss of vision.
AU-011 consists of Viral-like Nanoparticles that demonstrate highly selective targeting of solid tumours while leaving normal epithelium untouched. Aura’s lead product incorporates its viral-like nanoparticle conjugated with a potent cell-killing laser-activated molecule that is delivered efficiently and selectively to cancerous cells, thereby reducing or eliminating the risk of non-specific activity and undesirable toxicity. The molecule is activated by a standard ophthalmologic laser and treatment will be administered in an outpatient visit.
Aura Biosciences is applying nanotechnology to the fight against cancer. Its novel viral-like nanoparticle technology, developed in partnership with the National Cancer Institute (NCI), harnesses the potential of viral evolution and tumour targeting for the treatment of cancer.
