This designation comes after the FDA Office of Orphan Products Development reviewed recent Phase IIa clinical trial results, which showed both biological efficacy and safety in patients with the disease.
The US Orphan Drug Act promotes the creation of treatments for rare diseases, which are identified as conditions impacting fewer than 200,000 individuals nationwide.
The orphan designation offers multiple advantages, such as a seven-year period of market exclusivity following regulatory approval, exemption from FDA user fees for possible New Drug Application (NDA) submissions, eligibility to receive government grants for clinical trials, and access to specific tax credits.
Soligenix president and CEO Christopher Schaber said: “Behçet’s Disease is an area of unmet medical need, with up to 18,000 people in the US, 50,000 in Europe, 350,000 people in Turkey and as many as one million people worldwide affected by this incurable disease.
“Given the clinically meaningful improvements seen in a Phase II proof‑of‑concept study in patients with oral aphthous ulcers due to Behçet’s Disease, we are hopeful dusquetide will have a role to play in helping underserved patients suffering from this difficult to treat and chronic auto‑immune disease.
“The FDA’s decision to grant orphan drug designation to the SGX945 programme signifies an important step for Soligenix as we continue to advance the programme and adds significantly to the existing intellectual property estate surrounding this novel technology.”
Dusquetide helps to adjust the body’s response to injury and infection, fostering anti-inflammatory, anti-infective and tissue-healing processes.
In clinical trials involving humans, dusquetide demonstrated safety during a Phase I trial conducted with 84 volunteers. It also showed efficacy in Phase II and III studies that included over 350 participants suffering from oral mucositis while receiving chemoradiation for head and neck cancer.