— First and best FDA-approved treatment for the remedy of obtained hypothalamic weight problems, a unprecedented illness characterised by way of speeded up and sustained weight acquire brought about by way of hypothalamic damage or disorder –
— Indicated to cut back extra frame weight and deal with aid longer term in adults and pediatric sufferers elderly 4 years and older with obtained hypothalamic weight problems —
— Approval in keeping with -18.4% placebo-adjusted BMI aid accomplished by way of setmelanotide in world Segment 3 TRANSCEND trial [N=142] —
— Corporate to host convention name lately at 7:00 p.m. ET —
BOSTON, March 19, 2026 (GLOBE NEWSWIRE) — Rhythm Prescription drugs, Inc. (Nasdaq: RYTM), an international commercial-stage biopharmaceutical corporate considering reworking the lives of sufferers residing with uncommon neuroendocrine illnesses, lately introduced that the U.S. Meals and Drug Management (FDA) has permitted an expanded indication for IMCIVREE® (setmelanotide) to regard sufferers residing with obtained hypothalamic weight problems (HO).
Bought HO is a unprecedented illness characterised by way of speeded up and sustained weight acquire brought about by way of an damage to the hypothalamus or hypothalamic disorder. With this label enlargement, IMCIVREE is indicated to cut back extra frame weight and deal with aid longer term in adults and pediatric sufferers elderly 4 years and older with obtained 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, Leader Government 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 liable for controlling physiological purposes akin to power expenditure, starvation, and weight law. Bought HO maximum regularly follows tumors and their remedy or different hypothalamic damage or disorder. In response to research of the literature, tumor registries and claims information, Rhythm estimates there are roughly 10,000 other people residing with obtained HO within the U.S.
“Having a therapy for individuals and families affected by acquired hypothalamic obesity has the potential to be transformational,” stated Amy Wooden, Government 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 way of the sure pivotal Segment 3 TRANSCEND trial of setmelanotide in 142 sufferers with obtained HO. The worldwide find out about met its number one endpoint, with a statistically important -18.4% placebo-adjusted aid in frame mass index (BMI). For the main endpoint of imply BMI trade from baseline, find out about contributors on setmelanotide treatment (n=94) accomplished a -15.8% aid in comparison with a +2.6% build up amongst sufferers on placebo (n=48) at 52 weeks (p<0.0001). Setmelanotide was once usually smartly tolerated within the Segment 3 trial. The most typical opposed occasions (affecting >20% of contributors) 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) could also be permitted within the U.S. and Europe in grownup and pediatric sufferers elderly 2 years and older with syndromic or monogenic weight problems because of Bardet-Biedl syndrome (BBS) or Professional-opiomelanocortin (POMC), proprotein convertase subtilisin/kexin sort 1 (PCSK1), or leptin receptor (LEPR) deficiency.
Convention Name InformationRhythm will host a reside convention name and webcast at 7:00 p.m. ET lately to speak about the FDA approval of IMCIVREE for sufferers with obtained HO. To get entry to the reside convention name, contributors might sign in right here.
A reside webcast of the development will likely be to be had beneath “Events and Presentations” within the Investor Family members phase of the Rhythm Prescription drugs web page at https://ir.rhythmtx.com/. The archived webcast will likely be to be had on Rhythm Prescription drugs’ web page roughly two hours after the development and will likely be to be had for 30 days following the development.
About Bought Hypothalamic ObesityAcquired hypothalamic weight problems is a unprecedented illness characterised by way of speeded up and sustained weight acquire brought about by way of an damage to the hypothalamus. Hypothalamic damage might result in diminished alpha-melanocyte-stimulating hormone (α-MSH) manufacturing and impairment of MC4R pathway signaling. The MC4R pathway is liable for regulating power stability and frame weight. Bought hypothalamic weight problems maximum regularly follows the expansion or remedy of craniopharyngioma, astrocytoma or different hypothalamic-pituitary tumors. Further reasons of damage might come with stressful mind damage, stroke or irritation. Because of impairment of the MC4R pathway, sufferers enjoy speeded up and sustained weight acquire, incessantly accompanied by way of hyperphagia and/or diminished power expenditure. Bought hypothalamic weight problems can happen as early as six months following hypothalamic damage. Rhythm estimates there are roughly 10,000 other people residing with obtained HO within the U.S.
About Rhythm PharmaceuticalsRhythm is a commercial-stage biopharmaceutical corporate dedicated to remodeling the lives of sufferers and their households residing with uncommon neuroendocrine illnesses. Rhythm’s lead asset, IMCIVREE® (setmelanotide), an MC4R agonist designed to regard hyperphagia and critical weight problems, is permitted by way of the U.S. Meals and Drug Management (FDA) to cut back extra frame weight and deal with aid longer term in adults and pediatric sufferers elderly 4 years and older with obtained hypothalamic weight problems, grownup and pediatric sufferers 2 years of age and older with syndromic or monogenic weight problems because of Bardet-Biedl syndrome (BBS) or genetically showed pro-opiomelanocortin (POMC), together with proprotein convertase subtilisin/kexin sort 1 (PCSK1), deficiency or leptin receptor (LEPR) deficiency. Each the Ecu Fee (EC) and the United Kingdom’s Medications & Healthcare Merchandise Regulatory Company (MHRA) have approved setmelanotide for the remedy of weight problems and the keep watch over of starvation related to genetically showed BBS or genetically showed 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 extensive medical building 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 frame weight and deal with weight loss longer term in adults and pediatric sufferers elderly 4 years and older with obtained hypothalamic weight problems, in grownup and pediatric sufferers elderly 2 years and older with syndromic or monogenic weight problems because of Bardet-Biedl syndrome (BBS) or Professional-opiomelanocortin (POMC), proprotein convertase subtilisin/kexin sort 1 (PCSK1), or leptin receptor (LEPR) deficiency showed by way of genetic trying out demonstrating variants in POMC, PCSK1, or LEPR genes which can be interpreted as pathogenic, most likely pathogenic, or of unsure importance (VUS).
Within the Ecu Union and the UK, setmelanotide is indicated for the remedy of weight problems and the keep watch over of starvation related to genetically showed 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 Ecu Union and the UK, setmelanotide will have to be prescribed and supervised by way of a doctor with experience in weight problems with underlying genetic etiology.
Boundaries of UseSetmelanotide isn’t indicated for the remedy of sufferers with the next prerequisites as setmelanotide would now not be anticipated to be efficient:
Weight problems because of suspected POMC, PCSK1, or LEPR deficiency with POMC, PCSK1, or LEPR variants labeled as benign or most likely benignOther varieties of weight problems now not associated with obtained HO, BBS, or POMC, PCSK1 or LEPR deficiency, together with weight problems related to different genetic syndromes and basic (polygenic) weight problems.
Vital Protection Data
CONTRAINDICATIONSPrior critical hypersensitive reaction to setmelanotide or any of the excipients in IMCIVREE. Severe hypersensitive reaction reactions (e.g., anaphylaxis) were reported.
WARNINGS AND PRECAUTIONS
Disturbance in Sexual Arousal: Spontaneous penile erections and larger frequency of penile erections in men have took place. Tell sufferers that those occasions might happen and instruct sufferers who’ve an erection lasting longer than 4 hours to hunt emergency clinical consideration.
Melancholy and Suicidal Ideation: Melancholy and suicidal ideation have took place. Observe sufferers for brand new onset or worsening despair or suicidal ideas or behaviors. Imagine discontinuing IMCIVREE if sufferers enjoy suicidal ideas or behaviors, or clinically important or continual despair signs happen.
Allergy Reactions: Severe hypersensitive reaction reactions (e.g., anaphylaxis) were reported. If suspected, advise sufferers to promptly search clinical consideration and discontinue IMCIVREE.
Pores and skin Hyperpigmentation, Darkening of Pre-existing Nevi, and Construction of New Melanocytic Nevi: Generalized or focal will increase in pores and skin pigmentation took place within the majority of IMCIVREE-treated sufferers. IMCIVREE may additionally motive building of latest melanocytic nevi or darkening of pre-existing nevi. Carry out a complete frame pores and skin exam previous to initiation and periodically all over remedy to watch pre-existing and new pigmented lesions.
Acute Adrenal Insufficiency with Bought HO: Sufferers with obtained HO and secondary adrenal insufficiency reported critical opposed reactions associated with acute adrenal insufficiency in 5% of IMCIVREE-treated sufferers and no placebo-treated sufferers. In sufferers with secondary adrenal insufficiency, track for medical indicators of acute adrenal insufficiency.
Sodium Imbalance in Sufferers with Bought HO and Central Diabetes Insipidus: Sufferers with obtained 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 four% of placebo-treated sufferers. Observe serum sodium ranges with adjustments in fluid consumption and hydration standing. Regulate the doses of concomitant treatments for DI/AVP deficiency as wanted.
ADVERSE REACTIONSMost not unusual opposed reactions (prevalence ≥20% in no less than 1 indication) integrated pores and skin hyperpigmentation, injection website online reactions, nausea, headache, diarrhea, belly ache, vomiting, despair, and spontaneous penile erection.
USE IN SPECIFIC POPULATIONSTreatment with IMCIVREE isn’t really helpful when breastfeeding. Discontinue IMCIVREE when being pregnant is known until the advantages of treatment outweigh the possible dangers to the fetus.
To document SUSPECTED ADVERSE REACTIONS, touch Rhythm Prescription drugs at +1 (833) 789-6337 or FDA at 1-800-FDA-1088 or http://www.fda.gov/medwatch. See phase 4.8 of the Abstract of Product Traits for info on reporting suspected opposed reactions in Europe.
Please see the total Prescribing Data for added Vital Protection Data.
Ahead-Taking a look StatementsThis press unlock incorporates forward-looking statements inside the which means of the Personal Securities Litigation Reform Act of 1995. All statements contained on this press unlock that don’t relate to issues of ancient reality will have to be thought to be forward-looking statements, together with with out limitation statements in regards to the protection, efficacy, doable advantages of, and medical design or development of any of our merchandise or product applicants at any dosage or in any indication; the approval and use of IMCIVREE in sufferers with obtained hypothalamic weight problems and its availability for sufferers; the industrial enlargement of IMCIVREE; our expectancies surrounding doable regulatory submissions, development, or approvals and timing thereof for any of our product applicants; the estimated marketplace measurement and addressable inhabitants for our drug merchandise, together with IMCIVREE for the remedy of hypothalamic weight problems in the USA, the Ecu Union and Japan; the long run announcement of knowledge from our ongoing medical trials; the continued enrollment in and doable development or results of our medical trials; the presentation of the total information from the TRANSCEND find out about at an upcoming clinical assembly; and the content material, date and timing of any of the foregoing. Statements the usage of phrases akin to “expect”, “anticipate”, “believe”, “may”, “will” and identical phrases also are forward-looking statements. Such statements are matter to a lot of dangers and uncertainties, together with, however now not restricted to, our talent to sign up sufferers in medical trials, the design and consequence of medical trials, the affect of pageant, the facility to succeed in or download essential regulatory approvals, dangers related to information research and reporting, detrimental pricing laws, third-party repayment practices or healthcare reform projects, dangers related to the rules and laws governing our world operations and the prices of any similar compliance methods, our talent to effectively commercialize setmelanotide, our liquidity and bills, our talent to retain our key workers and experts, and to draw, retain and inspire certified team of workers, and basic financial prerequisites, and the opposite vital components, together with the ones mentioned beneath the caption “Risk Factors” in Rhythm’s Annual Document on Shape 10-Ok for the yr ended December 31, 2025 and our different filings with the Securities and Trade Fee. Excluding as required by way of legislation, we adopt no responsibilities to make any revisions to the forward-looking statements contained on this press unlock or to replace them to replicate occasions or instances happening after the date of this press unlock, whether or not because of new knowledge, long term tendencies or differently.
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