— First and solely FDA-approved remedy for the remedy of acquired hypothalamic weight problems, a uncommon illness characterised by accelerated and sustained weight acquire brought on by hypothalamic harm or dysfunction –
— Indicated to cut back extra physique weight and keep discount long run in adults and pediatric sufferers aged 4 years and older with acquired hypothalamic weight problems —
— Approval based mostly on -18.4% placebo-adjusted BMI discount achieved by setmelanotide in world Section 3 TRANSCEND trial [N=142] —
— Firm to host convention name at this time at 7:00 p.m. ET —
BOSTON, March 19, 2026 (GLOBE NEWSWIRE) — Rhythm Prescribed drugs, Inc. (Nasdaq: RYTM), a world commercial-stage biopharmaceutical firm centered on remodeling the lives of sufferers residing with uncommon neuroendocrine illnesses, at this time introduced that the U.S. Meals and Drug Administration (FDA) has accredited an expanded indication for IMCIVREE® (setmelanotide) to deal with sufferers residing with acquired hypothalamic weight problems (HO).
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Acquired HO is a uncommon illness characterised by accelerated and sustained weight acquire brought on by an harm to the hypothalamus or hypothalamic dysfunction. With this label growth, IMCIVREE is indicated to cut back extra physique weight and keep discount long run in adults and pediatric sufferers aged 4 years and older with acquired HO.
“IMCIVREE is now the first and only FDA-approved therapy for acquired HO, offering a targeted approach that addresses the underlying biology of this disease and meets a critical unmet need for patients who previously had no treatment options,” stated David Meeker, M.D., Chairman, Chief Govt Officer and President of Rhythm. “This is a transformative milestone for Rhythm and reinforces our commitment to bringing meaningful therapies to patients living with rare MC4R pathway diseases.”
The MC4R pathway is chargeable for controlling physiological features akin to power expenditure, starvation, and weight regulation. Acquired HO most ceaselessly follows tumors and their remedy or different hypothalamic harm or dysfunction. Primarily based on evaluation of the literature, tumor registries and claims information, Rhythm estimates there are roughly 10,000 folks residing with acquired HO within the U.S.
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“Having a therapy for individuals and families affected by acquired hypothalamic obesity has the potential to be transformational,” stated Amy Wooden, Govt Director and Founding father of the Raymond A. Wooden Basis. “We’ve seen firsthand the devastating impact acquired hypothalamic obesity has on patients’ and families’ lives, including relentless hunger and accelerated and sustained weight gain. IMCIVREE offers hope and a path forward for thousands of patients who have long been without options.”
The approval is supported by the constructive pivotal Section 3 TRANSCEND trial of setmelanotide in 142 sufferers with acquired HO. The worldwide research met its major endpoint, with a statistically vital -18.4% placebo-adjusted discount in physique mass index (BMI). For the first endpoint of imply BMI change from baseline, research individuals on setmelanotide remedy (n=94) achieved a -15.8% discount in contrast with a +2.6% enhance amongst sufferers on placebo (n=48) at 52 weeks (p<0.0001). Setmelanotide was typically effectively tolerated within the Section 3 trial. The commonest hostile occasions (affecting >20% of individuals) have been pores and skin hyperpigmentation, nausea, vomiting and headache.
“Setmelanotide has shown effectiveness in targeting the underlying biology of acquired HO,” stated Ashley Shoemaker, M.D., MSCI, Affiliate Professor of Pediatrics, Pediatric Endocrinology at Vanderbilt Well being. “Patients treated with setmelanotide experienced meaningful reductions in BMI and hunger, demonstrating the therapy’s ability to deliver clinically significant outcomes in both children and adult patients. Acquired HO is a severe disease that requires early and proactive management. With the availability of IMCIVREE, physicians can offer a targeted therapy.”
IMCIVREE® (setmelanotide) can also be accredited within the U.S. and Europe in grownup and pediatric sufferers aged 2 years and older with syndromic or monogenic weight problems as a result of Bardet-Biedl syndrome (BBS) or Professional-opiomelanocortin (POMC), proprotein convertase subtilisin/kexin kind 1 (PCSK1), or leptin receptor (LEPR) deficiency.
Convention Name InformationRhythm will host a stay convention name and webcast at 7:00 p.m. ET at this time to debate the FDA approval of IMCIVREE for sufferers with acquired HO. To entry the stay convention name, individuals could register right here.
A stay webcast of the occasion shall be accessible beneath “Events and Presentations” within the Investor Relations part of the Rhythm Prescribed drugs web site at https://ir.rhythmtx.com/. The archived webcast shall be accessible on Rhythm Prescribed drugs’ web site roughly two hours after the occasion and shall be accessible for 30 days following the occasion.
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About Acquired Hypothalamic ObesityAcquired hypothalamic weight problems is a uncommon illness characterised by accelerated and sustained weight acquire brought on by an harm to the hypothalamus. Hypothalamic harm could result in decreased alpha-melanocyte-stimulating hormone (α-MSH) manufacturing and impairment of MC4R pathway signaling. The MC4R pathway is chargeable for regulating power steadiness and physique weight. Acquired hypothalamic weight problems most ceaselessly follows the expansion or remedy of craniopharyngioma, astrocytoma or different hypothalamic-pituitary tumors. Further causes of harm could embody traumatic mind harm, stroke or irritation. Because of impairment of the MC4R pathway, sufferers expertise accelerated and sustained weight acquire, usually accompanied by hyperphagia and/or decreased power expenditure. Acquired hypothalamic weight problems can happen as early as six months following hypothalamic harm. Rhythm estimates there are roughly 10,000 folks residing with acquired HO within the U.S.
About Rhythm PharmaceuticalsRhythm is a commercial-stage biopharmaceutical firm dedicated to reworking the lives of sufferers and their households residing with uncommon neuroendocrine illnesses. Rhythm’s lead asset, IMCIVREE® (setmelanotide), an MC4R agonist designed to deal with hyperphagia and extreme weight problems, is accredited by the U.S. Meals and Drug Administration (FDA) to cut back extra physique weight and keep discount long run in adults and pediatric sufferers aged 4 years and older with acquired hypothalamic weight problems, grownup and pediatric sufferers 2 years of age and older with syndromic or monogenic weight problems as a result of Bardet-Biedl syndrome (BBS) or genetically confirmed pro-opiomelanocortin (POMC), together with proprotein convertase subtilisin/kexin kind 1 (PCSK1), deficiency or leptin receptor (LEPR) deficiency. Each the European Fee (EC) and the UK’s Medicines & Healthcare Merchandise Regulatory Company (MHRA) have approved setmelanotide for the remedy of weight problems and the management of starvation related to genetically confirmed BBS or genetically confirmed loss-of-function biallelic POMC, together with PCSK1, deficiency or biallelic LEPR deficiency in adults and youngsters 2 years of age and above. Moreover, Rhythm is advancing a broad scientific growth program for setmelanotide in different uncommon illnesses, in addition to investigational MC4R agonists bivamelagon and RM-718, and a preclinical suite of small molecules for the remedy of congenital hyperinsulinism. Rhythm’s headquarters is in Boston, MA.
Setmelanotide IndicationIn the USA, setmelanotide is indicated to cut back extra physique weight and keep weight discount long run in adults and pediatric sufferers aged 4 years and older with acquired hypothalamic weight problems, in grownup and pediatric sufferers aged 2 years and older with syndromic or monogenic weight problems as a result of Bardet-Biedl syndrome (BBS) or Professional-opiomelanocortin (POMC), proprotein convertase subtilisin/kexin kind 1 (PCSK1), or leptin receptor (LEPR) deficiency confirmed by genetic testing demonstrating variants in POMC, PCSK1, or LEPR genes which are interpreted as pathogenic, doubtless pathogenic, or of unsure significance (VUS).
Within the European Union and the UK, setmelanotide is indicated for the remedy of weight problems and the management of starvation related to genetically confirmed BBS or loss-of-function biallelic POMC, together with PCSK1, deficiency or biallelic LEPR deficiency in adults and youngsters 2 years of age and above. Within the European Union and the UK, setmelanotide ought to be prescribed and supervised by a doctor with experience in weight problems with underlying genetic etiology.
Limitations of UseSetmelanotide will not be indicated for the remedy of sufferers with the next circumstances as setmelanotide wouldn’t be anticipated to be efficient:
Weight problems as a result of suspected POMC, PCSK1, or LEPR deficiency with POMC, PCSK1, or LEPR variants labeled as benign or doubtless benignOther forms of weight problems not associated to acquired HO, BBS, or POMC, PCSK1 or LEPR deficiency, together with weight problems related to different genetic syndromes and common (polygenic) weight problems.
Essential Security Info
CONTRAINDICATIONSPrior severe hypersensitivity to setmelanotide or any of the excipients in IMCIVREE. Critical hypersensitivity reactions (e.g., anaphylaxis) have been reported.
WARNINGS AND PRECAUTIONS
Disturbance in Sexual Arousal: Spontaneous penile erections and elevated frequency of penile erections in males have occurred. Inform sufferers that these occasions could happen and instruct sufferers who’ve an erection lasting longer than 4 hours to hunt emergency medical consideration.
Despair and Suicidal Ideation: Despair and suicidal ideation have occurred. Monitor sufferers for brand new onset or worsening despair or suicidal ideas or behaviors. Take into account discontinuing IMCIVREE if sufferers expertise suicidal ideas or behaviors, or clinically vital or persistent despair signs happen.
Hypersensitivity Reactions: Critical hypersensitivity reactions (e.g., anaphylaxis) have been reported. If suspected, advise sufferers to promptly search medical consideration and discontinue IMCIVREE.
Pores and skin Hyperpigmentation, Darkening of Pre-existing Nevi, and Improvement of New Melanocytic Nevi: Generalized or focal will increase in pores and skin pigmentation occurred within the majority of IMCIVREE-treated sufferers. IMCIVREE might also trigger growth of recent melanocytic nevi or darkening of pre-existing nevi. Carry out a full physique pores and skin examination previous to initiation and periodically throughout remedy to watch pre-existing and new pigmented lesions.
Acute Adrenal Insufficiency with Acquired HO: Sufferers with acquired HO and secondary adrenal insufficiency reported severe hostile reactions associated to acute adrenal insufficiency in 5% of IMCIVREE-treated sufferers and no placebo-treated sufferers. In sufferers with secondary adrenal insufficiency, monitor for scientific indicators of acute adrenal insufficiency.
Sodium Imbalance in Sufferers with Acquired HO and Central Diabetes Insipidus: Sufferers with acquired HO and concomitant central diabetes insipidus (DI)/arginine vasopressin (AVP) deficiency reported hyponatremia in 6% of IMCIVREE-treated sufferers and a pair of% of placebo-treated sufferers and hypernatremia in 5% of IMCIVREE-treated sufferers and 4% of placebo-treated sufferers. Monitor serum sodium ranges with adjustments in fluid consumption and hydration standing. Alter the doses of concomitant therapies for DI/AVP deficiency as wanted.
ADVERSE REACTIONSMost widespread hostile reactions (incidence ≥20% in at the very least 1 indication) included pores and skin hyperpigmentation, injection web site reactions, nausea, headache, diarrhea, belly ache, vomiting, despair, and spontaneous penile erection.
USE IN SPECIFIC POPULATIONSTreatment with IMCIVREE will not be advisable when breastfeeding. Discontinue IMCIVREE when being pregnant is acknowledged except the advantages of remedy outweigh the potential dangers to the fetus.
To report SUSPECTED ADVERSE REACTIONS, contact Rhythm Prescribed drugs at +1 (833) 789-6337 or FDA at 1-800-FDA-1088 or http://www.fda.gov/medwatch. See part 4.8 of the Abstract of Product Traits for data on reporting suspected hostile reactions in Europe.
Please see the complete Prescribing Info for extra Essential Security Info.
Ahead-Trying StatementsThis press launch accommodates forward-looking statements throughout the which means of the Non-public Securities Litigation Reform Act of 1995. All statements contained on this press launch that don’t relate to issues of historic reality ought to be thought of forward-looking statements, together with with out limitation statements concerning the security, efficacy, potential advantages of, and scientific design or progress of any of our merchandise or product candidates at any dosage or in any indication; the approval and use of IMCIVREE in sufferers with acquired hypothalamic weight problems and its availability for sufferers; the business development of IMCIVREE; our expectations surrounding potential regulatory submissions, progress, or approvals and timing thereof for any of our product candidates; the estimated market dimension and addressable inhabitants for our drug merchandise, together with IMCIVREE for the remedy of hypothalamic weight problems in the USA, the European Union and Japan; the long run announcement of information from our ongoing scientific trials; the continuing enrollment in and potential progress or outcomes of our scientific trials; the presentation of the complete information from the TRANSCEND research at an upcoming medical assembly; and the content material, date and timing of any of the foregoing. Statements utilizing phrases akin to “expect”, “anticipate”, “believe”, “may”, “will” and comparable phrases are additionally forward-looking statements. Such statements are topic to quite a few dangers and uncertainties, together with, however not restricted to, our capacity to enroll sufferers in scientific trials, the design and final result of scientific trials, the influence of competitors, the power to attain or get hold of vital regulatory approvals, dangers related to information evaluation and reporting, unfavorable pricing laws, third-party reimbursement practices or healthcare reform initiatives, dangers related to the legal guidelines and laws governing our worldwide operations and the prices of any associated compliance applications, our capacity to efficiently commercialize setmelanotide, our liquidity and bills, our capacity to retain our key staff and consultants, and to draw, retain and encourage certified personnel, and common financial circumstances, and the opposite necessary elements, together with these mentioned beneath the caption “Risk Factors” in Rhythm’s Annual Report on Kind 10-Ok for the yr ended December 31, 2025 and our different filings with the Securities and Trade Fee. Besides as required by regulation, we undertake no obligations to make any revisions to the forward-looking statements contained on this press launch or to replace them to replicate occasions or circumstances occurring after the date of this press launch, whether or not because of new data, future developments or in any other case.
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