Pegcetacoplan (Aspaveli), a new medicine for people with rare kidney conditions C3 glomerulopathy (C3G) and immune complex membranoproliferative glomerulonephritis (IC-MPGN), is not recommended as an NHS-funded treatment following a NICE (National Institute for Health and Care Excellence) draft recommendation published Friday 13 March.
The recommendation, which may change following a public consultation and further consideration by NICE, recognises that pegcetacoplan reduces the amount of protein in the urine and slows the decline of kidney function. However, the NICE committee felt there were too many uncertainties when trying to calculate whether the treatment’s value for money fell within NICE’s limits.
This is hugely disappointing news and Kidney Care UK urge NICE to reverse its decision in the final guidance. This would be the first recommended treatment that targets the underlying disease and delays progression, rather than just dealing with symptoms.
We are working closely with the MPGN/DDD Support Group to ensure that the voice of people affected by these conditions is heard by NICE. The effectiveness of the treatment is accepted by NICE, so we are hopeful that if additional analysis can be carried out to better understand the cost-effectiveness it will be possible for this group of patients to access this vital drug.
NICE will consider responses to the draft guideline consultation and publish final guidance later in spring. You can comment on the guideline on the NICE website.
These are devastating kidney conditions that affect mainly children and young people. Their lives, and those of their families, are full of challenges as they navigate a life-changing illness which for many will lead to kidney failure requiring dialysis or a transplant. We have a treatment that can change that future and it’s crucial that a way is found to make it available on the NHS.Samantha Sharp, Senior Policy Officer, Kidney Care UK
C3 glomerulopathy (C3G) is an immune system disorder that causes damage to the tiny filters in the kidney (glomeruli). It occurs when part of the immune system called complement C3 is deposited in the kidneys. This affects the kidneys’ ability to filter the blood and can eventually lead to a reduction of kidney function. C3G is a subtype of a condition called membranoproliferative glomerulonephritis (MPGN). Immune complex membranoproliferative glomerulonephritis (IC-MPGN) is a related condition, also caused by the immune system damaging the kidneys.
Pegcetacoplan aims to treat the underlying disease, by regulating overactivity of the complement system, part of the body’s immune system. Clinical trials have shown that pegcetacoplan reduces protein in the urine and slows the decline of kidney function compared with placebo.
Do you or somebody in your family have C3G or IC-MPGN? Kidney Care UK has created a survey to collect information about people's experiences of these conditions and their treatments.
Please complete our anonymous survey, which will contribute to our response to NICE.