FDA Grants Orphan Status to Diabetes Reversal Drug
- By BSTQ Staff
DiaVacs’ type 1 diabetes therapy DV-0100 has been granted orphan drug designation by the U.S. Food and Drug Administration (FDA). The therapy uses a proprietary technology that is designed to halt the body’s autoimmune reaction against the pancreatic islet cells that are responsible for producing insulin, allowing them to produce insulin normally and reversing the trajectory of the disease. It does this by taking the patient’s own dendritic cells from their blood, modifying the cells through the use of small interfering oligonucleotides, and then vaccinating the patient with these modified cells under the skin with a small needle. The cells are absorbed and trafficked to the pancreatic lymph nodes, thereby inducing tolerance.
The therapy has been shown to be safe and effective in animal models of type 1 diabetes, and there has been no evidence of safety signals in human Phase I trials in patients with established type 1 diabetes for five years or longer. In some patients in the Phase I human trials, production of endogenous insulin was measured even after 10 years of disease. The company has initiated an FDA-approved Phase II human trial.
Orphan designation qualifies DV-0100 for seven years of marketing exclusivity in the U.S. if the company is the first to obtain marketing approval for this product in type 1 diabetes. It also qualifies the company for certain tax credits and waivers for prescription drug user fees.
