Rhythm Pharmaceuticals Announces NICE Guidance for IMCIVREE® (Setmelanotide) Updated to Include Adults Living with Bardet-Biedl Syndrome
Existing NHS funding arrangements for children ages two to five in England and Wales remain unchanged.
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Rhea-AI Summary
Rhythm Pharmaceuticals (Nasdaq: RYTM) announced expanded NICE guidance allowing adults with genetically confirmed Bardet-Biedl syndrome to access funded IMCIVREE treatment. The National Institute for Health and Care Excellence recommendation expands reimbursement eligibility through the National Health Service. The updated recommendation covers adults in England, Wales and Northern Ireland.
The guidance broadens NICE's May 2024 recommendation, which had limited treatment initiation to patients ages 6 to 17 years. NICE considered additional clinical, real-world and disease-burden evidence, including longer-term treatment data. Its assessment recognized the burden of excessive hunger and obesity associated with Bardet-Biedl syndrome, including effects on quality of life, mental health, mobility, education and independence.
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Positive
- Moderate pointExpanded NICE reimbursement eligibility gives adults with genetically confirmed Bardet-Biedl syndrome access to NHS-funded IMCIVREE.
Negative
- None.
News Explained
NICE’s new guidance expands funded adult access, while leaving the existing NHS England specialized-commissioning arrangements for children ages two to five with BBS in England and Wales unchanged.
Details
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Key Figures
- Age range in prior NICE recommendation
- 6 to 17 years
- Previous recommendation for setmelanotide in BBS
Key Terms
hyperphagia medical
mc4r agonist medical
anaphylaxis medical
biallelic medical
AI-generated analysis. How Rhea-AI works. Not financial advice.
-- Expanded guidance based on additional evidence demonstrating the benefits of IMCIVREE in adults living with Bardet-Biedl syndrome --
BOSTON, Nov. 04, 2026 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (Nasdaq: RYTM), a global commercial-stage biopharmaceutical company focused on transforming the lives of patients living with rare neuroendocrine diseases, today announced that the National Institute for Health and Care Excellence (NICE) has issued a positive guidance for IMCIVREE® (setmelanotide) to include adults living with genetically confirmed Bardet-Biedl syndrome (BBS).
The expansion of reimbursement eligibility by NICE allows adults living with BBS to access funded treatment through the National Health Service (NHS). The updated guidance expands NICE's previous recommendation for setmelanotide in BBS from May 2024, which had optimized treatment initiation for patients ages 6 to 17 years. NICE considered additional clinical, real-world and disease burden evidence, including longer-term treatment data, and recognized the significant burden that hyperphagia and obesity associated with BBS can have on patients and families. This assessment reflects the broad impact of BBS on quality of life, mental health, mobility, education and independence.
"This updated recommendation is welcome news for adults in England, Wales and Northern Ireland living with Bardet-Biedl syndrome," said Professor Philip Beales, Emeritus Professor of Molecular Genetics and Genomic Medicine at University College London. "BBS is a lifelong disease, and adults continue to experience the significant burden of hyperphagia and obesity throughout their lives. Expanding access to setmelanotide means more people living with BBS will have the opportunity to receive treatment.”
Setmelanotide for children between the ages of two to five years old in England and Wales with BBS is funded through NHS England specialized commissioning arrangements. The updated NICE guidance does not change existing access arrangements for this group.
This press release is intended for investors only.
About Rhythm Pharmaceuticals
Rhythm is a commercial-stage biopharmaceutical company committed to transforming the lives of patients and their families living with rare neuroendocrine diseases. Rhythm’s lead asset, IMCIVREE® (setmelanotide), an MC4R agonist designed to treat hyperphagia and severe obesity, is approved by the U.S. Food and Drug Administration (FDA) to reduce excess body weight and maintain weight reduction long term in adult and pediatric patients aged 4 years and older with acquired hypothalamic obesity, adult and pediatric patients 2 years of age and older with syndromic or monogenic obesity due to Bardet-Biedl syndrome (BBS) or genetically confirmed pro-opiomelanocortin (POMC), including proprotein convertase subtilisin/kexin type 1 (PCSK1), deficiency or leptin receptor (LEPR) deficiency. Both the European Commission (EC) and the UK’s Medicines and Healthcare Products Regulatory Agency (MHRA) have authorized setmelanotide for the treatment of obesity and control of hunger in patients 4 years of age and above with acquired hypothalamic obesity due to hypothalamic injury or impairment; and for the treatment of obesity and the control of hunger associated with genetically confirmed BBS or genetically confirmed loss-of-function biallelic POMC, including PCSK1, deficiency or biallelic LEPR deficiency in adults and children 2 years of age and above. Additionally, Rhythm is advancing a broad clinical development program for setmelanotide in other rare diseases, as well as investigational MC4R agonists bivamelagon and RM-718, and a preclinical suite of small molecules for the treatment of congenital hyperinsulinism. Rhythm’s headquarters is in Boston, MA.
Setmelanotide Indication
In the United States, setmelanotide is indicated to reduce excess body weight and maintain weight reduction long term in adults and pediatric patients aged 4 years and older with acquired hypothalamic obesity, in adult and pediatric patients aged 2 years and older with syndromic or monogenic obesity due to Bardet-Biedl syndrome (BBS) or Pro-opiomelanocortin (POMC), proprotein convertase subtilisin/kexin type 1 (PCSK1), or leptin receptor (LEPR) deficiency confirmed by genetic testing demonstrating variants in POMC, PCSK1, or LEPR genes that are interpreted as pathogenic, likely pathogenic, or of uncertain significance (VUS).
In the European Union and the United Kingdom, setmelanotide is indicated for the treatment of obesity and the control of hunger associated with genetically confirmed BBS or loss-of-function biallelic POMC, including PCSK1, deficiency or biallelic LEPR deficiency in adults and children 2 years of age and above, and for the treatment of obesity and control of hunger in adults and children 4 years of age and above with acquired hypothalamic obesity due to hypothalamic injury or impairment. In the European Union and the United Kingdom, setmelanotide should be prescribed and supervised by a physician experienced in the diagnosis and management of rare forms of obesity with genetic or hypothalamic origin.
The European Union summary of product characteristics can be accessed by clinking this LINK.
The United Kingdom summary of product characteristics can be access by clicking this LINK.
Information for audiences in the Unites States of America
Limitations of Use
Setmelanotide is not indicated for the treatment of patients with the following conditions as setmelanotide would not be expected to be effective:
- Obesity due to suspected POMC, PCSK1, or LEPR deficiency with POMC, PCSK1, or LEPR variants classified as benign or likely benign
- Other types of obesity not related to acquired HO, BBS, or POMC, PCSK1 or LEPR deficiency, including obesity associated with other genetic syndromes and general (polygenic) obesity.
Important Safety Information
CONTRAINDICATIONS
Prior serious hypersensitivity to setmelanotide or any of the excipients in IMCIVREE. Serious hypersensitivity reactions (e.g., anaphylaxis) have been reported.
WARNINGS AND PRECAUTIONS
Disturbance in Sexual Arousal: Spontaneous penile erections and increased frequency of penile erections in males have occurred. Inform patients that these events may occur and instruct patients who have an erection lasting longer than 4 hours to seek emergency medical attention.
Depression and Suicidal Ideation: Depression and suicidal ideation have occurred. Monitor patients for new onset or worsening depression or suicidal thoughts or behaviors. Consider discontinuing IMCIVREE if patients experience suicidal thoughts or behaviors, or clinically significant or persistent depression symptoms occur.
Hypersensitivity Reactions: Serious hypersensitivity reactions (e.g., anaphylaxis) have been reported. If suspected, advise patients to promptly seek medical attention and discontinue IMCIVREE.
Skin Hyperpigmentation, Darkening of Pre-existing Nevi, and Development of New Melanocytic Nevi: Generalized or focal increases in skin pigmentation occurred in the majority of IMCIVREE-treated patients. IMCIVREE may also cause development of new melanocytic nevi or darkening of pre-existing nevi. Perform a full body skin examination prior to initiation and periodically during treatment to monitor pre-existing and new pigmented lesions.
Acute Adrenal Insufficiency with Acquired HO: Patients with acquired HO and secondary adrenal insufficiency reported serious adverse reactions related to acute adrenal insufficiency in
Sodium Imbalance in Patients with Acquired HO and Central Diabetes Insipidus: Patients with acquired HO and concomitant central diabetes insipidus (DI)/arginine vasopressin (AVP) deficiency reported hyponatremia in
ADVERSE REACTIONS
Most common adverse reactions (incidence ≥
USE IN SPECIFIC POPULATIONS
Treatment with IMCIVREE is not recommended when breastfeeding. Discontinue IMCIVREE when pregnancy is recognized unless the benefits of therapy outweigh the potential risks to the fetus.
To report suspected adverse reactions in the United States, contact Rhythm Pharmaceuticals at +1 (833) 789-6337 or FDA at 1-800-FDA-1088 or http://www.fda.gov/medwatch.
By reporting side effects, you can help provide more information on the safety of this medicine
Please see the full Prescribing Information for additional Important Safety Information.
Forward-looking Statements
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including, without limitation, statements regarding the safety, efficacy, potential benefits of, and clinical design or progress of any of our products or product candidates at any dosage or in any indication, including, the impact of certain regulatory approvals of IMCIVREE for the treatment of BBS in England, Wales and Northern Ireland and expectations surrounding coverage and availability of IMCIVREE; and the timing of any of the foregoing. Statements using words such as “expect”, “anticipate”, “believe”, “may”, “will” and similar terms are also forward-looking statements. Such statements are subject to numerous risks and uncertainties, including, but not limited to, our ability to enroll patients in clinical trials, the design and outcome of clinical trials, the impact of competition, the ability to achieve or obtain necessary regulatory approvals, risks associated with data analysis and reporting, unfavorable pricing regulations, third-party reimbursement practices or healthcare reform initiatives, risks associated with the laws and regulations governing our international operations and the costs of any related compliance programs, our ability to successfully commercialize setmelanotide, our liquidity and expenses, our ability to retain our key employees and consultants, and to attract, retain and motivate qualified personnel, and general economic conditions, and the other important factors, including those discussed under the caption “Risk Factors” in Rhythm’s Quarterly Report on Form 10-Q for the three months ended June 30, 2026, and our other filings with the Securities and Exchange Commission. Except as required by law, we undertake no obligations to make any revisions to the forward-looking statements contained in this release or to update them to reflect events or circumstances occurring after the date of this release, whether as a result of new information, future developments or otherwise.
Corporate Contacts:
David Connolly
Head of Investor Relations and Corporate Communications
Rhythm Pharmaceuticals, Inc.
857-264-4280
dconnolly@rhythmtx.com
Kate Walsh
Director, Corporate Communications
Rhythm Pharmaceuticals, Inc.
(857) 264-4280
kwalsh@rhythmtx.com
FAQ
AI-generated questions and answers. How Rhea-AI works. Not financial advice.
What does the updated NICE guidance mean for Rhythm Pharmaceuticals' IMCIVREE?
Adults with genetically confirmed Bardet-Biedl syndrome can now access NHS-funded IMCIVREE under the expanded NICE guidance. The recommendation extends access beyond the May 2024 guidance, which had limited treatment initiation to patients ages 6 to 17 years.
Does the updated IMCIVREE NICE guidance change access for children ages two to five?
Existing access arrangements remain unchanged for children ages two to five with Bardet-Biedl syndrome in England and Wales. Setmelanotide treatment for this group is funded through NHS England specialized commissioning arrangements.