Joe Todisco is chief executive officer of CorMedix and spoke to OSP about how the company’s product, DefenCath, could put an end to the additional misery dialysis patients often go through by being at higher risk for and developing serious infections.
He says there are 800,000 people in the U.S. who are living with end-stage kidney disease with more than half of those belonging to an ethnic or minority group. And while some of those with the disease will have a transplant, most will have dialysis – a treatment that filters waste from the kidneys and removes excess fluid from the body.
The company's product, DefenCath, is a formulation of taurolidine and heparin with broad antimicrobial activity that is currently being investigated for use as a catheter lock solution, with the aim of reducing the risk of catheter-related bloodstream infections from central venous catheters.
Todisco explained that dialysis means that patients’ bloodstreams are frequently accessed using needles but also catheters - soft plastic tubes inserted into larger veins.
Critical unmet need
He said: “There are these catheter-related bloodstream infections which are a critical unmet medical need. At the moment there is no drug product that is FDA approved or in development for the prevention of these infections other than us.
“With access required as frequently as it is for patients on dialysis, this automatically increases the risk of infection and in some cases can be more serious leading to sepsis and in worst case scenarios, death. There are several germs that can cause infections with the most common being staph (staphylococcus aureus) and that can be deadly.”
DefenCath has been designated by FDA as Fast Track and as a Qualified Infectious Disease Product (QIDP), and the original New Drug Application (NDA) received priority review in recognition of its potential to address an unmet medical need."
QIDP provides for an additional five years of marketing exclusivity, which will be added to the five years granted to a New Chemical Entity. CorMedix also committed to conducting a clinical study in pediatric patients using a central venous catheter for hemodialysis which will add an additional six months of marketing exclusivity when the study is completed.
Todisco said: “We had to go to them with multiple pathways and one was a highly accelerated pathway that would have allowed us to go to market within 60 days. They did not fully agree with the accelerated pathway, but they did say, let’s combine your one and two pathways.”
He said the company’s current manufacturer of heparin still had some hurdles to jump from a compliance standpoint and that the FDA wanted to assess their corrective actions. The FDA told CorMedix to submit both data packages which they will review in parallel over the next six months.
He added: “The FDA said if they can’t get comfortable with the compliance of the original API source, they would just tell us to withdraw it and then the other pathway will have already be reviewed by then too. We thought that was a great compromise from the FDA.
“We are expecting to get an acceptance for review by next week (June 15) and we think we’re going to have a target action date on the application for mid-November. We won’t know our target date until the FDA accepts the NDA for review which should be mid-June and if it is, we think we are going to have a target action date on the application for mid-November. By and large we were very happy with the FDA’s discussion and the data they’ve requested – we had everything they asked for ready.”
Follow-up inspection
He said the company was making sure the manufacturers were ready for a follow-up inspection and the other was preparing for commercial launch.
“Those are the two things we now have going on simultaneously – obviously we have no control over the FDA, and we don’t own our manufacturer but we believe they’re going to be ready and we don’t see any major obstacles right through to getting approval later this year so hopefully we can launch in early 2024.”
The product is a proprietary formulation of taurolidine 1.35%, citrate 3.5% and heparin 1000 units/mL that is currently being investigated for use as a catheter lock solution, with the aim of reducing the risk of infections from in-dwelling catheters.
Taurolidine, the key compound is an amino acid derivative with in-vitro studies indicating broad antimicrobial activity against gram-positive and gram-negative bacteria, including antibiotic resistant strains, as well as mycobacteria and clinically relevant fungi including Aspergillus.
Dialysis - incredibly disruptive
Liz Masson-Hurlburt is executive vice president, head of clinical and medical affairs.
She said: “Dialysis is already incredibly disruptive and what we have done is come up with one drug that has the potential to rule out a number of infections symptomatic of hemodialysis and the various risk factors within that. It really is unpleasant enough having to have a catheter and the last thing people need on top of that is any sort of infection, let alone a serious one.
“What we are trying to do is bring a unique, cost-effective and straightforward solution to the market.”
Patients receiving dialysis are vulnerable and Todisco points out they have high rates of mortality.
He added: “We are in a position to have a meaningful impact on this patient population and hopefully improve their quality of life and reverse some of these trends.
“Hopefully we can reduce hospitalizations, reduce length of stay – these are all things we want to see once we get the product into commercial use."
