Melbourne-based AdAlta (ASX:1AD) has received Orphan Drug Designation from the FDA for AD-114, a novel first-in-class drug candidate for the treatment of idiopathic pulmonary fibrosis (IPF).
In a statement, the company said there is growing global interest in new treatments for fibrosis, especially for idiopathic pulmonary fibrosis, which has a 50 to 70 per cent mortality rate and affects 135,000 people in the US every year.
"Current therapies for the treatment of IPF are sub-optimal and there is a high-unmet medical need," it said. "AD-114 has strong pre-clinical results for IPF, demonstrating both anti-fibrotic and anti-inflammatory activity in human lung tissue and indicating greater efficacy than existing approved IPF drugs."
Orphan Drug status provides R&D tax credits, new drug application fee waivers and a seven year period of market exclusivity from the US FDA following approval.
According to AdAlta CEO Sam Cobb, "Securing Orphan Designation for our lead program represents an important step in our strategy for the rapid commercial development of AD-114 for the treatment of idiopathic pulmonary disease. We expect this orphan designation, when combined with our pre-clinical package of AD-114, will help accelerate our commercial development path.
“AdAlta remains on track to meet its stated clinical development milestones and management remains focused on expediting AD-114 into Phase 1 human clinical trials for idiopathic pulmonary fibrosis by early 2018.”
AdAlta said it also demonstrated broad fibrotic application of AD-114, demonstrating the potential of AD-114 for the treatment of other fibrotic diseases including the liver (NASH) and eye (wet-AMD).