Rienso: Withdrawal of the application to change the marketing authorisation

Rienso is a medicine already used to treat patients with iron-deficiency anaemia associated with chronic kidney disease (long-term, progressive decrease in the ability of the kidneys to work properly).

Iron deficiency is common in anaemic patients with long-term kidney disease, and can be due to many factors including the poor absorption of dietary iron from food.

Rienso has been authorised since June 2012. It is available as a solution for infusion (drip) into a vein and contains the active substance ferumoxytol.

Rienso was also expected to be used for iron-deficiency anaemia in patients with conditions other than long-term kidney disease, when iron given by mouth is ineffective or inappropriate or where rapid restoration of iron levels is needed.

Lack of iron reduces the body's ability to make haemoglobin, the oxygen-carrying pigment in the blood, resulting in anaemia. The active substance in Rienso, ferumoxytol, is an iron-containing compound. After injection, it is taken up by cells in the liver, spleen and the bone marrow, and releases iron that replaces the body's depleted iron stores. With the iron stores replenished, the body can produce more haemoglobin, which will help correct the anaemia.

The applicant presented data from two main studies involving 1,413 patients with iron deficiency anaemia of various causes. In one study Rienso was compared with placebo (a dummy treatment) and in the other with another injectable iron compound used to treat anaemia, iron sucrose. The main measure of effectiveness was how much haemoglobin levels rose 5 weeks after start of treatment. The company also provided other supporting data, including analyses of the safety of the medicine.

The application was withdrawn after the CHMP had evaluated the documentation provided by the company and formulated lists of questions. After the CHMP had assessed the company's responses to the questions, there were still some unresolved issues.

Based on the review of the data and the company's response to the CHMP lists of questions, at the time of the withdrawal, the CHMP considered that Rienso had been shown to be more effective than placebo and at least as effective as iron sucrose in treating iron-deficiency anaemia associated with various causes other than long-term kidney disease. However, the Committee had concerns about the safety of the medicine in this wider group of patients, particularly as post-marketing reports of serious or fatal hypersensitivity (allergic) reactions have been noted in regular ongoing safety monitoring. It was not possible to be certain of the extent of the risk from the submitted information.

Therefore, at the time of the withdrawal, the CHMP considered that the benefits of Rienso in the treatment of iron-deficiency anaemia in this extended population did not outweigh its risks and was of the provisional opinion that Rienso could not have been approved for the treatment of anaemia due to iron deficiency of any cause.

In its letter notifying the Agency of the withdrawal of application, the company stated that it had withdrawn the application because the CHMP considered that further data were needed to support a wider use of the medicine.

The company informed the CHMP that there are no consequences for patients who are receiving Rienso for iron-deficiency anaemia in clinical trials or compassionate use programmes.

If you are in a clinical trial or compassionate use programme and need more information about your treatment, contact the doctor who is giving it to you.

There are no consequences of this withdrawal on the use of Rienso in its authorised indication.