The National Institute for Health and Care Excellence (NICE) have issued final draft guidance recommending the use of the first licensed treatment for primary immunoglobulin A nephropathy (IgAN).
The drug is called targeted-release budesonide and is recommended as an option for treatment for IgAN where there is risk of rapid disease progression in adults with a urine protein-to-creatinine ratio of 1.5g/g or more.
Guy Hill, a member of Kidney Care UK’s Patient Advisory group, was a patient expert during the NICE appraisal. He participated in NICE committee meetings, making sure that the committee understood how the condition affects people and the importance of preventing disease progression.
Guy said: “Targeted-release budesonide is a really exciting drug for IgAN sufferers, who have had no effective drugs before that can slow down the causation of extra IgA in the blood stream that goes on to damage the kidney. Unlike the inconclusive trials of the steroid prednisolone before, the reported side effects in these budesonide trials are minimal and it does not stop the efficiency of IgA working in the immune system around the rest of the body. The proposed dosage for people with IgAN is initially 9 months treatment and then further dosages if the IgA returns.
"IgAN is such a cruel disease with an average diagnosis age of 40. This means so many young people find this disease so destabilising as they take on life from employment to relationships and having families. If it does lead to kidney failure, then people are faced with a lifetime of NHS treatment. To have a drug which can slow the decline of the disease is fantastic.”
Fiona Loud, Policy Director at Kidney Care UK, commented: “Although not a cure, the hope is that the use of targeted-release budesonide may help many people with IgAN who have slow declining kidney function (80% of people with the condition) from ever reaching kidney failure. It may also mean that those who have the more aggressive type of IgAN (around 20% of people with the condition) may have a slower journey to kidney failure. This is the first time that a treatment that specifically targets IgA production has been recommended. Excitingly, if combined with the new SLGT2 drugs, the disease progression that people with IgAN have could be very different from those in the past.”
Targeted-release budesonide is recommended only if it is an add-on to optimised standard care including the highest tolerated licensed dose of angiotensin-converting enzyme (ACE) inhibitors or angiotensin-receptor blockers (ARBs), unless those treatments are not recommended for an individual.
The final draft guidance has been sent to all groups involved in the appraisal who have 15 working days to consider whether they wish to appeal against it. Subject to any appeal by consultees, the draft guidance may be used as the basis for NICE's guidance on the use of the treatment in the NHS in England. Final guidance is expected to be published in December 2023.
We recommend you speak to your clinician if you have any questions about this treatment.