Perth-based Dimerix (ASX:DXB) has received Orphan Designation from the US FDA for propagermanium and irbesartan, the constituent parts of its drug candidate DMX-200 for the treatment of a condition known as focal segmental glomerulosclerosis (FSGS), a leading cause of chronic kidney disease.
There are no therapies currently approved by the FDA for FSGS, according to the company.
Patients with FSGS, who progress to kidney failure and receive a kidney transplant, have a 30 to 40 per cent chance of the condition reccurring.
The Orphan Drug Designation program provides orphan status to drugs which are defined as those intended for the safe and effective treatment, diagnosis or prevention of rare diseases and disorders that affect fewer than 200,000 people in the US, or that affect more than 200,000 persons but are not expected to recover the costs of developing and marketing a treatment drug.
Orphan Drug Designation provides the company with seven-year FDA-administered market Orphan Drug Exclusivity (ODE) on successful approval of the drug. It also qualifies the company for various regulatory and financial support measures as the treatment progresses through pre-clinical and clinical development in the US.
According to Dimerix Executive Chairman, Dr James Williams, “Securing Orphan Designation for our lead program represents an important step in our strategy for the rapid commercial development of DMX-200 for the treatment of chronic kidney disease. We expect this orphan designation, when combined with pending data from our current Phase II trial in Australia, will help accelerate our current clinical and commercial development path in the important US medical market.”