A world-first international trial of hepcidin to regulate iron absorption in haemochromatosis patients is underway, including sites in Brisbane, Sydney and Melbourne.

The Phase 2 trial aims to compare weekly dosing of a synthetic human hepcidin versus placebo on markers of iron metabolism.

If effective at preventing iron overload, the treatment may be a useful alternative to removing excess iron via regular venesection.

Hepatologist Professor Darrell Crawford, from the University of Queensland and Gallipoli Medical Research Institute, told the limbic there were certainly people who had difficulty complying with venesection protocols.

“Despite being a widely available, safe and effective treatment, it’s surprising how poor compliance can be – either from difficult access to veins or through general compliance issues.”

“And it may be that some patients will find a drug is an acceptable alternative.”

He said iron-chelating agents in the past had been difficult to administer and had a lot of side effects.

“The new agents are more tolerable but nowhere near as effective in removing iron as venesection therapy.”

Hence the opportunity for a new approach to keep iron stores low and prevent the risk of developing complications such as cirrhosis and primary liver cancer.

“We know in haemochromatosis the basic defect is a deficiency of this protein so this trial is giving hepcidin to people who have established haemochromatosis who have been on venesection to look at whether it is a potential alternative therapy or supplement to venesection.”

“And we know that is important, because early recognition, early diagnosis and early therapy of haemochromatosis can restore life expectancy to normal.”

The study is only recruiting about 60 patients who will have weekly visits. Patients in the active arm of the trial will receive a weekly subcutaneous injection of hepcidin for 12 weeks.

“How the therapeutic dosing will pan out in the long run, depends upon the results from this early study,” Professor Crawford said.

“The patients that we have on trial to date tolerate the treatment very well.”

He said it was important that Australian sites were part of the global trial.

“Australia has made an enormous contribution to our understanding of haemochromatosis. A lot of the basic and clinical research related to disorders of iron metabolism has come from Australians. And in fact the very first paper describing this hepcidin deficiency in humans came from Brisbane.”

Meanwhile, the Pharmaceutical Benefits Advisory Committee (PBAC) has recommended Section 100 (Highly Specialised Drugs Program) listing of a new form of deferasirox for the treatment of chronic iron overload due to disorders of haemopoiesis.

The PBAC’s recommendation for listing was based on its assessment that deferasirox film coated tablets were biocomparable, rather than bioequivalent, to deferasirox dispersible tablets and a cost-minimisation analysis between the film coated and dispersible tablets.

Deferasirox are available in 90mg, 180mg and 360mg tablets.

At its July meeting, the PBAC also recommended an unrestricted listing of ferric derisomaltose for the treatment of iron deficiency anaemia when treatment with oral iron is ineffective or not tolerated.

This recommendation was made on a cost-minimisation basis with the main comparator, ferric carboxymaltose.

The injection is available as 500 mg iron in 5 ml.