Drug development company Dimerix (ASX: DXB) has confirmed that the European Commission (EC) has approved its DMX-200 drug to be used as an “orphan medicinal product” for the treatment of Focal Segmental Glomerulosclerosis (FSGS), a form of chronic kidney disease that is currently the leading cause of kidney failure in adults globally.
Orphan designation is typically granted for drugs that intend to assist life-threatening or debilitating diseases whereby patients have essentially run out of treatment options.
Such drugs are put into the hands of clinicians, to be provided as experimental new therapies that have the potential to significantly benefit patients and possibly improve their quality of life and chance of survival.
Dimerix said that the EC’s approval follows a “positive opinion” in October from the European Medicine Agency’s (EMA) Committee for Orphan Medicinal Products (COMP).
According to the EC, receiving Orphan Drug Designation (ODD) in Europe for FSGS means that Dimerix has established that “the intention to treat FSGS with DMX-200 was justified based on preliminary non-clinical data which showed a reduction in the number of podocytes lost and an improvement in proteinuria.”
Furthermore, the EMA has determined that Dimerix has “provided sufficient justification for the assumption that DMX-200 will be of significant benefit to those affected by the condition,” and that, as an alternative to currently marketed products, “this constitutes a clinically relevant advantage”.
Gradual progress for DMX-200
Orphan drug designation is a significant advance in the development of DMX-200 for Dimerix.
The designation carries with it regulatory and financial benefits that could expedite DMX-200 being marketed in Europe, including reduced fees during the product development phase, direct access to centralised marketing authorisation and possibly most importantly, 10-year market exclusivity following product approval.
Today’s news also means that Dimerix has now achieved ODD status in both Europe and the US after DMX-200 was granted ODD from the United States Food and Drug Administration (FDA) in December 2015.
In a bid to advance DMX-200 in the US, Dimerix is currently enrolling patients for two distinct Phase 2 clinical programs – DMX-200 for FSGS and DMX-200 for Diabetic Kidney Disease. According to Dimerix, patients with diabetic kidney disease showed a “clinically and statistically significant efficacy response” in an initial Phase 2a clinical study carried out last year.
With the studies now in full swing, Dimerix has declared that subject to patient recruitment rates, preliminary results are anticipated for both studies in Q4 2019.
Just last week, Dimerix “dosed” the first patient in its diabetic kidney disease study at the Renal Research in Gosford, New South Wales.
The study intends to enrol 40 eligible patients across Australia, who will receive two treatment blocks of 12 weeks during which they will receive either placebo or DMX-200, taken as an oral capsule.
“The European Commission’s decision to grant orphan drug designation to DMX-200 for the treatment of FSGS is a key regulatory milestone that will further drive our clinical development program,” said Dr Nina Webster, CEO of Dimerix.
“We believe that DMX-200, which has already received orphan drug designation for the treatment of FSGS from the FDA in the United States, has the potential to provide a much-needed innovative therapeutic option for patients who have been diagnosed with FSGS,” said Dr Webster.