NICE Requests More Information on Alexion’s Soliris Off-Label Use

The UK’s healthcare watchdog says it needs more evidence before it can determine if off-label use of Alexion’s Soliris is effective in preventing the recurrence of a rare kidney disease in patients who have undergone transplants.

In an evidence summary published last week, the National Institute for Health and Care Excellence notes that the cost for a five-week treatment of Soliris (eculizumab) for transplant patients who have C3 glomerulopathy is $79,000 and the annual maintenance cost is more than $500,000. 

Because of the rarity of the disease, only 10 case study reports exist involving patients who have had kidney transplants. While seven of the reports conclude Soliris improved or stabilized the condition in patients, one showed a partial response and two were ineffective. Additionally, the evidence shows no case reports where Soliris prevented the recurrence of C3 glomerulopathy, according to NICE.

The disease occurs in only one or two people per million. Patients face a 30 percent risk of end-stage renal disease after two years, and 70 percent of those undergoing kidney transplantation risk recurrence, while graft loss is more than 50 percent.

In its summary, NICE called for longer, larger, statistically powered and adequately controlled studies to better evaluate Soliris’ effect on patient survival, graft survival, adverse effects and quality of life in the post-transplant population.

An Alexion spokeswoman said NICE undertook its study after receiving a request from the doctor of a 12-year-old girl who suffers from the disease. The girl is set to receive a kidney transplant, but hopes of obtaining Soliris were dashed when the National Health Service declined to pay for her treatment.

The NHS is expected to rule on the off-label use in a few weeks, the spokeswoman said.
Soliris is approved in nearly 50 countries for patients with paroxysmal nocturnal hemoglobinuria, a debilitating blood disorder characterized by destruction of red blood cells. It is also approved in nearly 40 countries to treat patients with atypical hemolytic uremic syndrome, a life-threatening disease that causes abnormal blood clots to form in small vessels in the kidneys.

Read the NICE evidence summary at www.fdanews.com/06-30-15-soliris.pdf. — John Bechtel