Feraheme: Withdrawal of the marketing authorisation application

Feraheme was developed for the treatment of iron deficiency anaemia (IDA, when a lack of iron in the body leads to low levels of haemoglobin and red blood cells) in patients who cannot take iron supplements by mouth or for whom iron supplements do not work well enough. It was also intended for use by patients with IDA due to chronic kidney disease, as these patients may not produce enough red blood cells.

Feraheme contains the active substance ferumoxytol and was to be available as a solution for infusion (drip) into a vein.

The active substance in Feraheme, ferumoxytol, is an iron-containing compound. When injected into the blood, it is taken up by cells in the liver, spleen and bone marrow, where the iron is released from the compound and replenishes the body’s low iron stores. With the increased iron stores, the body can produce more haemoglobin, which will help correct the anaemia.

The company presented the results of three main studies conducted in patients with iron deficiency anaemia due to chronic kidney disease and two main studies conducted in patients with iron deficiency anaemia due to other causes who could not take iron supplements by mouth or for whom iron supplements did not work. The main measure of effectiveness was the change in the blood level of haemoglobin in patients given Feraheme compared with that in patients given other iron preparations or placebo (a dummy treatment). The company also submitted supportive data on effectiveness and data on the safety of Feraheme from other studies.

The application was withdrawn after the European Medicines Agency had evaluated the initial information from the company and had prepared questions for the company. The company had not responded to the questions at the time of the withdrawal.

As results of the main studies showed that Feraheme was effective at treating iron deficiency anaemia due to different causes, the Agency considered that the proposed use of the medicine should cover all patients with iron deficiency anaemia, without differentiating between anaemia due to kidney disease and anaemia due to other causes.

In addition, the Agency raised a number of questions about uncertainties regarding the effectiveness and safety of Feraheme, to which the company would have needed to provide satisfactory answers before Feraheme could be authorised. At the time of the withdrawal, as the company had not yet responded to the questions, the Agency’s opinion was that the benefits of Feraheme did not outweigh its risks.

In its letter notifying the Agency of the withdrawal of the application, the company stated that it was doing so for business reasons.

The company informed the Agency that there are no consequences for patients in clinical trials using Feraheme.

If you are in a clinical trial and need more information about your treatment, speak with your clinical trial doctor.