MOLECULAR INFORMATION
Molecular information
- Type
- MC4R-selective melanocortin receptor agonist · daily subcutaneous
Setmelanotide is a selective MC4R agonist developed by Rhythm Pharmaceuticals, authorised by the EMA under Imcivree for genetic obesities caused by POMC, PCSK1 or LEPR deficiency, and Bardet-Biedl syndrome. The first peptide to treat monogenic obesity rather than the polygenic kind.
Indications (EU authorisation)
- Obesity due to POMC, PCSK1 or LEPR deficiency (confirmed by genetic test)
- Bardet-Biedl syndrome — obesity component
Why we grade it A
EMA-authorised with robust Phase 3 data in narrow, genetically-confirmed populations. Not promoted as a general weight-loss agent; the orphan indication scope is preserved in labelling. Grade A rather than S because the indication is narrow and the long-term outcome data is still maturing.
RECONSTITUTION
How to reconstitute
YOU WILL NEED
- Bacteriostatic water (BAC water)
- Insulin syringes (0.5–1 mL)
- Alcohol swabs
- Vial of lyophilized peptide
- Sterile work surface
PROCEDURE
- 01Clean the work area and hands thoroughly.
- 02Calculate the required bacteriostatic-water volume for the desired concentration.
- 03Draw the calculated BAC water into the syringe.
- 04Inject the water slowly down the inside wall of the vial — not directly onto the powder cake.
- 05Gently swirl until fully dissolved. Never shake.
- 06Store the reconstituted vial refrigerated at 2–8 °C and use within 28 days.
Standard biotech reconstitution procedure. Always use sterile technique. This is research-protocol guidance, not medical advice.
References
- POMC / LEPR Phase 321 participantsBody weight change in POMC/LEPR-deficient obesity — 80% of POMC participants achieved ≥10% weight loss at 1 year
- Bardet-Biedl Phase 338 participantsBBS-associated obesity — 32% mean BMI reduction at 1 year
Frequently asked questions
- What is Setmelanotide used for?
- An orphan-indication treatment for genetic obesity caused by POMC, PCSK1 or LEPR deficiency, and obesity in Bardet-Biedl syndrome. Confirmation of the genetic variant by clinical genetic testing is required before authorisation applies.
- Is Setmelanotide authorised in the EU?
- Yes — EMA-authorised since 2021 under Imcivree for the narrow genetic-obesity indications. It is not authorised for general weight management.
- Who makes Setmelanotide?
- Rhythm Pharmaceuticals (US), with EU distribution via established medical-products channels. The drug is high-cost orphan therapy, dispensed through specialist clinics.
- Why is Setmelanotide not used for general weight loss?
- Its mechanism (MC4R agonism) is the same one disrupted in the genetic deficiencies it treats. In the general obese population the pathway is functional, so the benefit-risk balance hasn't been established outside the genetic indications.