NICE publishes evidence summary on eculizumab (Soliris) for treating rare kidney condition

29 Jun 2015 12:58 PM

NICE has completed a rapid evidence summary on the off-label use of eculizumab (Soliris, Alexion Pharma UK) for managing dense deposit disease (DDD), a form of the rare kidney condition C3 glomerulopathy.

Although there was no evidence for the use of eculizumab after a kidney transplant to prevent the disease recurring, there were a small number of cases where the drug was used to stop the progression of the disease when it had recurred. 

The NICE evidence summary considers the published international evidence of eculizumab’s effectiveness, safety, cost and patient factors in determining whether prophylactic use of eculizumab is effective and safe for preventing recurrence of C3 glomerulopathy after kidney transplantation. It is not formal NICE guidance but is intended to inform NHS England’s decision on the use of the drug to prevent recurrence of dense deposit disease in people who have had a kidney transplant.

Dense deposit disease (DDD) is a type of C3 glomerulopathy and is characterised by dense deposits of C3 proteins in the kidneys as a result of an underlying genetic or acquired abnormality of the body’s complement immune system.

It damages the tiny filters in the kidneys preventing them from correctly filtering waste from the blood. More than half of people with DDD will go on to develop end stage renal disease requiring kidney dialysis and eventual kidney transplantation within 10 years of diagnosis.

The risk of the disease coming back after kidney transplantation is also high - over 70% - and more than half of people will lose their transplanted kidney as a result.

NICE recently recommended eculizumab for funding for treating adults and children with atypical haemolytic uraemic syndrome (aHUS). Eculizumab also has a marketing authorisation in the UK for treating or paroxysmal nocturnal haemoglobinuria (PNH). Like C3 glomerulopathy, aHUS and PNH are complement-mediated diseases, which stimulated interest in using eculizumab to treat this condition.

The evidence summary looks specifically at the use of eculizumab to prevent recurrence of DDD C3 glomerulopathy in people who have had a kidney transplant because of their disease. It highlights that there are currently no studies or case reports of eculizumab to prevent recurrence of DDD C3 glomerulopathy after a kidney transplant.

However, a small open-label study of 6 people, 3 of whom had had a kidney transplant, and 7 reports of single cases who had recurrence of C3 glomerulopathy post-transplant and were using eculizumab to prevent progression of the disease were found.

Although eculizumab prevented progression of C3 glomerulopathy in the majority of cases, only a partial response was seen in 1 case and it was ineffective in 2 cases. In addition, it is possible that cases in which eculizumab has been unsuccessful are under reported. Vaccination against meningococcal infection is essential before and during treatment with eculizumab. However, vaccination may not be sufficient to prevent infection. The evidence summary concludes that more robust studies are needed to better evaluate eculizumab for preventing recurrence of C3 glomerulopathy post-transplant.

Paul Chrisp, Programme Director for the Medicines and Prescribing Centre at NICE, said: “In this rapid review, we looked through hundreds of pieces of research from across the world. There was no research looking at using eculizumab to stop this disease coming back after a kidney transplant, but there were cases that suggested it could be useful in stopping the disease progressing once it had returned.

“While a licensed medicine meets acceptable standards of efficacy, safety, and quality, clinical situations will arise where the use of unlicensed medicines or the use of medicines to treat conditions for which they are not licensed may be judged by the prescriber to be in the best interest of the patient. This is particularly the case for certain rare diseases, such as DDD, where there are often not enough patients to enlist on the clinical trials that are needed to develop a drug.

“In these situations it is extremely important that any decision to use an unlicensed or off-label medicine is made on the basis of the best available evidence of safety and efficacy and whether the medicine is more or less likely to improve outcomes for individual patients. NICE evidence summaries, while not formal guidance, help to ensure that the decisions of clinicians and the choices made by patients are properly informed and made on the basis of the best available evidence.”

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