NICE extends setmelanotide for Bardet-Biedl syndrome to people who start treatment as adults
The drug was previously recommended only for people who began it between the ages of 6 and 17. The NHS in England must now fund it for adults within 90 days where it is the most suitable option.
Adults with Bardet-Biedl syndrome (BBS) in England will be able to start treatment with setmelanotide on the NHS, after the National Institute for Health and Care Excellence (NICE) widened its recommendation on Wednesday 4 November.
The new guidance covers obesity and hyperphagia linked to genetically confirmed BBS in anyone aged 6 and over. For people aged 18 and over, the NHS in England must fund the drug within 90 days of the guidance being published, where it is considered the most suitable treatment. It should already be funded for 6 to 17-year-olds. Children aged 2 to 5 can also get it, in line with NHS England's commissioning position.
The decision replaces NICE's earlier guidance from 22 May 2024, which recommended setmelanotide only for people who started it between 6 and 17, while allowing them to carry on into adulthood. Anyone already being treated outside the new recommendation can continue until they and their clinician decide to stop.
The condition
BBS is a rare genetic disorder caused by changes in one or more BBS genes, of which 22 have been identified. Those genes are involved in a signalling system in the hypothalamus that regulates hunger, fullness and how the body uses energy. When it is disrupted, people can experience hyperphagia, which NICE describes as a feeling similar to starvation, leading to severe obesity that begins in early childhood.
Other features can include learning difficulties, sight loss, kidney problems, extra fingers or toes, and genital or hormonal problems. NICE's committee heard that blindness is common by the mid-teenage years, and that hunger linked to BBS is often mistaken by healthcare professionals for ordinary hunger.
For adults who did not start setmelanotide before 18, the usual care until now has been best supportive care, built around dietary restriction and exercise.
Trial evidence and cost
Clinical trial results suggest setmelanotide may reduce weight and body mass index in people aged 6 and over with BBS. Hunger scores and quality of life improved in the short term, though NICE notes hunger scores may not reliably reflect changes in hyperphagia. Updated evidence suggests the effect on weight may last longer term, which the committee also judged uncertain.
Across children and adults together, the cost-effectiveness estimates came in below the range NICE normally treats as an acceptable use of NHS resources, which is why it can be used routinely.
Setmelanotide is made by Rhythm Pharmaceuticals under the brand name Imcivree and is given by injection. Its list price is £2,376.00 for a 10 mg/ml vial, excluding VAT, and the company supplies it to the NHS at a confidential discount under a commercial arrangement.
