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The guidance used to stop at 17. Adults with this rare condition are now included

Qurexa Editorial Team5 November 20267 min read 0 0
The guidance used to stop at 17. Adults with this rare condition are now included

What happened

On 4 November 2026, the National Institute for Health and Care Excellence - NICE - updated its guidance on a medicine called setmelanotide, sold as Imcivree. The change is small in wording and large in effect. The guidance now covers adults with genetically confirmed Bardet-Biedl syndrome. Until now, NHS funding in England, Wales and Northern Ireland followed a recommendation made in May 2024, which covered people who started treatment between the ages of 6 and 17. That created an odd situation. A child could start the medicine at 15 and keep taking it. But an adult who was never offered it as a child could not start it at all. The update removes that gap. The medicine's maker, Rhythm Pharmaceuticals, announced the decision and said NICE had looked at extra evidence, including clinical data, real-world data, data on how much the condition affects people's lives, and longer-term information on people who had stayed on treatment. Professor Philip Beales, Emeritus Professor of Molecular Genetics and Genomic Medicine at University College London, called the update "welcome news" for adults in England, Wales and Northern Ireland, and pointed out that Bardet-Biedl syndrome is a lifelong condition. One group is unaffected. Children aged two to five in England and Wales already receive setmelanotide through NHS England specialised commissioning arrangements. This update does not change that.

Why this matters

Bardet-Biedl syndrome is rare. It is a genetic condition that can affect many parts of the body, including sight, kidneys and learning. Two of its most difficult features are obesity and something called hyperphagia. Hyperphagia is not the same as having a big appetite. It is a constant, powerful hunger that does not switch off after eating. People describe it as never feeling full. It is driven by a faulty signal in the brain, not by willpower or habit, and that distinction matters enormously. This is why the old age limit stung. Hyperphagia does not stop at 18. An adult living with that hunger every day, who had grown up before the medicine existed or whose diagnosis came late, could watch a younger person be offered treatment and be told they were not eligible. It also matters because of how rare conditions are usually treated by health systems. Medicines for very small groups of patients are expensive per person, and they are often approved slowly, in narrow bands, or not at all. A decision that widens access, rather than narrowing it, is relatively unusual. For families affected by Bardet-Biedl syndrome, this is the kind of news that has been hoped for since 2024.

What the evidence actually says

It is worth being careful here, because the announcement came from the company that makes the medicine, and company announcements naturally emphasise good news. What is solid: NICE has published updated guidance, and it does now include adults. NICE's own published guidance list for 4 November 2026 includes setmelanotide for obesity and hyperphagia in Bardet-Biedl syndrome for people aged 6 years and over. That is the official record, and it is what determines NHS funding. What the announcement did not include: any new efficacy figures from the NICE assessment. The press release described the types of evidence NICE considered, but it did not publish numbers showing how much weight adults lost or how much their hunger reduced. So we cannot tell you from this announcement how well it works in adults specifically. What we can tell you about side effects comes with an important caveat. The safety figures in the company's release are drawn from prescribing information for a different condition - acquired hypothalamic obesity - not from the Bardet-Biedl assessment. With that caveat, those figures were: acute adrenal insufficiency in 5% of treated patients compared with 0% on placebo; low blood sodium in 6% compared with 2%; and high blood sodium in 5% compared with 4%. The most common side effects, affecting at least one in five people in at least one indication, were darkening of the skin, reactions where the injection is given, nausea, headache, diarrhoea, tummy pain, vomiting, depression, and spontaneous erections. Some of those are mild. Some are not. Adrenal insufficiency can be serious and needs medical attention. Depression as a listed side effect is something to discuss honestly with a specialist before starting. Setmelanotide is a daily injection under the skin, not a tablet. That is a real commitment, every day, for a condition that lasts a lifetime.

Qurexa perspective

This is a medicine people inject themselves, at home, every single day, for years. That is exactly the situation where adherence support earns its place. We support people with medication adherence, and the pattern we see with daily treatments is consistent: the first few weeks are usually fine, and the difficulty comes later, when the routine has to survive holidays, illness, a change of job, or simply a bad week. Specialist medicines also often need careful storage and reliable, on-time supply, because a gap is not easily filled at short notice. If you or someone in your family starts setmelanotide, it is worth setting up the practical side deliberately rather than hoping it sorts itself out: a fixed time of day, a reminder that actually works for you, somewhere safe and correctly stored for the medicine, and a reorder habit that leaves a buffer rather than running to the last dose. We would add one honest caution. Medicines for rare conditions are usually supplied through specialist NHS centres rather than ordinary community routes, so your specialist team, not a general delivery service, will be your main point of contact for the medicine itself.

Practical advice

If you are an adult with Bardet-Biedl syndrome, or you care for someone who is, here is where to start. Speak to your specialist team, not your GP, in the first instance. Bardet-Biedl syndrome is managed through specialist genetic and endocrine services, and they are the people who can assess whether setmelanotide is right for you and arrange it. Check that your diagnosis is genetically confirmed. The guidance applies to people with genetically confirmed Bardet-Biedl syndrome. If you were diagnosed clinically years ago and never had genetic testing, that is worth raising, because it may affect eligibility. Ask directly about the side effects listed above, especially adrenal insufficiency and mood. Ask what monitoring you would have, how often, and what warning signs should make you seek help urgently. Ask what happens if it does not work for you. NICE recommendations for this kind of medicine often include rules about stopping treatment if there is not enough benefit. Knowing those rules in advance is better than discovering them later. If you live in Scotland, this decision does not apply to you. NICE guidance covers England, Wales and Northern Ireland. Scotland has its own process through the Scottish Medicines Consortium, so ask your specialist about access there. Do not buy this medicine online. It is a prescription-only specialist treatment that needs monitoring, and unregulated supplies are both unsafe and a waste of money.

What to know

NICE has updated its guidance so that adults with genetically confirmed Bardet-Biedl syndrome can now get NHS-funded setmelanotide in England, Wales and Northern Ireland. Previously, funding effectively required treatment to start between the ages of 6 and 17. Children aged two to five in England and Wales already had access through separate NHS England arrangements, and that has not changed. The condition causes relentless hunger, called hyperphagia, that is driven by brain signalling rather than willpower. That is why a medicine, rather than diet advice alone, can help. Be realistic about what we know. The announcement came from the manufacturer and did not include new effectiveness figures for adults. The side-effect data quoted came from a different condition's prescribing information. The treatment is a daily injection with real side effects, including some that need monitoring. All of that is worth discussing properly with a specialist. Sources: GlobeNewswire / Rhythm Pharmaceuticals, 'Rhythm Pharmaceuticals Announces NICE Guidance for IMCIVREE (Setmelanotide) Updated to Include Adults Living with Bardet-Biedl Syndrome', 4 November 2026, https://www.globenewswire.com/news-release/2026/11/04/3395223/0/en/rhythm-pharmaceuticals-announces-nice-guidance-for-imcivree-setmelanotide-updated-to-include-adults-living-with-bardet-biedl-syndrome.html | NICE, 'Published guidance, NICE advice and quality standards', updated 4 November 2026, https://www.nice.org.uk/guidance/published | NICE, 'Setmelanotide for treating obesity and hyperphagia in Bardet-Biedl syndrome (HST31)', May 2024, https://www.nice.org.uk/guidance/hst31/chapter/1-Recommendations This article is for general information and does not replace advice from a doctor, pharmacist or other qualified healthcare professional.

#NICE#Bardet-Biedl syndrome#rare diseases#setmelanotide#NHS treatment#obesity

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