Rhythm Pharmaceuticals Announces FDA Approval of IMCIVREE® (setmelanotide) for Patients with Acquired Hypothalamic Obesity

Q: Who is eligible for IMCIVREE under the new RYTM FDA indication and when will it be available?

IMCIVREE is indicated for adults and pediatric patients aged 4 years and older with acquired hypothalamic obesity. According to the company, IMCIVREE will be available for U.S. patients immediately with Rhythm InTune support.

Q: What safety issues were observed in the TRANSCEND trial for setmelanotide (RYTM)?

The most common adverse events (>20%) were skin hyperpigmentation, nausea, vomiting and headache. According to the company, setmelanotide was generally well tolerated in the Phase 3 trial.

Rhea-AI Impact

(High)

Rhea-AI Sentiment

(Neutral)

Rhea-AI Summary

Rhythm Pharmaceuticals (NASDAQ: RYTM) announced FDA approval of IMCIVREE (setmelanotide) for acquired hypothalamic obesity (HO) in adults and pediatric patients aged 4 years and older. Approval was based on the Phase 3 TRANSCEND trial showing a -18.4% placebo-adjusted BMI reduction.

IMCIVREE is available immediately in the U.S.; Rhythm estimates ~10,000 people in the U.S. live with acquired HO. The company will host a conference call today at 7:00 p.m. ET.

Positive

First and only FDA-approved therapy for acquired hypothalamic obesity
-18.4% placebo-adjusted BMI reduction in Phase 3 TRANSCEND (N=142)
Indicated for adults and pediatrics aged 4+, expanding eligible patient population
Immediate U.S. availability with patient support program Rhythm InTune

Negative

Common adverse events (>20%) include skin hyperpigmentation, nausea, vomiting, headache
Phase 3 TRANSCEND size (N=142) may limit subgroup precision for diverse patient groups

News Market Reaction – RYTM

-3.17%

9 alerts

-3.17% News Effect

+11.7% Peak in 16 hr 46 min

-$202M Valuation Impact

$6.18B Market Cap

0.9x Rel. Volume

On the day this news was published, RYTM declined 3.17%, reflecting a moderate negative market reaction. Argus tracked a peak move of +11.7% during that session. Our momentum scanner triggered 9 alerts that day, indicating moderate trading interest and price volatility. This price movement removed approximately $202M from the company's valuation, bringing the market cap to $6.18B at that time.

Data tracked by StockTitan Argus on the day of publication.

Key Figures

Placebo-adjusted BMI change: -18.4% TRANSCEND sample size: 142 patients BMI reduction on setmelanotide: -15.8% +5 more

8 metrics

Placebo-adjusted BMI change -18.4% Primary result from Phase 3 TRANSCEND trial in acquired HO

TRANSCEND sample size 142 patients Global Phase 3 TRANSCEND trial in acquired HO

BMI reduction on setmelanotide -15.8% Mean BMI change from baseline at 52 weeks (n=94)

BMI change on placebo +2.6% Mean BMI change from baseline at 52 weeks (n=48)

P-value p<0.0001 Primary endpoint BMI change at 52 weeks

Age eligibility (HO) 4 years and older Approved indication for acquired hypothalamic obesity

Age eligibility (other indications) 2 years and older Existing IMCIVREE approvals in BBS and certain monogenic obesities

Estimated U.S. HO population 10,000 patients Company estimate for acquired hypothalamic obesity in the U.S.

Market Reality Check

Price: $88.12 Vol: Volume 993,669 is 13% abo...

normal vol

$88.12 Last Close

Volume Volume 993,669 is 13% above the 20-day average of 882,457. normal

Technical Price $90.31 is trading below the 200-day MA of $96.07 despite a 5.18% gain.

Peers on Argus

RYTM gained 5.18% on the FDA approval while close peers showed mixed moves (e.g....

1 Up

RYTM gained 5.18% on the FDA approval while close peers showed mixed moves (e.g., CYTK +1.53%, LEGN -1.43%). Only one peer (CORT) appeared in momentum scans, suggesting a stock-specific reaction rather than a broad biotech move.

Historical Context

5 past events · Latest: Mar 16 (Negative)

Pattern 5 events

Date	Event	Sentiment	Move	Catalyst
Mar 16	Phase 3 EMANATE data	Negative	-3.1%	Phase 3 EMANATE topline failed to meet primary endpoints in four substudies.
Feb 26	Earnings and pipeline	Positive	-1.1%	Reported strong 2025 IMCIVREE revenue and cash plus sNDA PDUFA date disclosure.
Feb 17	Earnings date set	Neutral	+4.5%	Announced timing of Q4/FY2025 earnings call and a TD Cowen conference appearance.
Feb 05	Conference participation	Neutral	-3.8%	Disclosed participation in Guggenheim biotech summit with a planned fireside chat.
Jan 09	Prelim revenues, milestones	Positive	+3.6%	Prelim IMCIVREE revenue growth and 2026 clinical/milestone roadmap including HO sNDA PDUFA.

Pattern Detected

News has often moved RYTM, but reactions to positive updates are mixed, with some strong revenue and pipeline milestones followed by modest pullbacks or only moderate gains.

Recent Company History

Over the last few months, Rhythm has highlighted strong IMCIVREE growth, with preliminary Q4 and FY2025 product revenues and a PDUFA date of March 20, 2026 for acquired HO. Subsequent earnings confirmed $57.3M Q4 and $194.8M full-year 2025 revenue, alongside substantial cash. The company then reported EMANATE Phase 3 genetic obesity results that did not meet primary endpoints, which weighed on shares. Today’s FDA approval for acquired hypothalamic obesity builds directly on prior TRANSCEND data and the previously disclosed PDUFA timeline.

Regulatory & Risk Context

Active S-3 Shelf · $200,000,000

Shelf Active

Active S-3 Shelf Registration 2026-02-26

$200,000,000 registered capacity

An effective S-3ASR shelf dated 2026-02-26 registers resale of 2,395,831 common shares from preferred conversion and permits the company to sell up to $200,000,000 of common stock via an at-the-market agreement with TD Securities (USA) LLC. No usage has been reported yet, but the facility allows future issuances via prospectus supplements.

Market Pulse Summary

This announcement highlights FDA approval of IMCIVREE for acquired hypothalamic obesity based on the...

Analysis

This announcement highlights FDA approval of IMCIVREE for acquired hypothalamic obesity based on the Phase 3 TRANSCEND trial, which showed a placebo-adjusted BMI reduction of −18.4% across 142 patients with a highly significant p<0.0001. The label expansion adds to existing indications in rare genetic obesities and targets an estimated 10,000 U.S. patients. Investors may track launch execution, safety profile in broader use, and any future use of the company’s $200,000,000 ATM facility.

Key Terms

acquired hypothalamic obesity, body mass index (BMI), phase 3, adverse events, +4 more

8 terms

acquired hypothalamic obesity medical

"for patients with acquired hypothalamic obesity, a rare disease characterized..."

A condition in which damage to the brain’s hypothalamus—often from surgery, injury, or a tumor—disrupts the body’s internal “thermostat” for hunger, metabolism and energy use, causing rapid, hard-to-control weight gain. Investors care because it creates a clearly defined patient group with high unmet medical need, predictable clinical outcomes, and potential demand for therapies or devices; progress or setbacks in treating this condition can drive valuation swings for companies developing targeted treatments.

body mass index (BMI) medical

"based on -18.4% placebo-adjusted BMI reduction achieved by setmelanotide..."

Body mass index (BMI) is a simple number calculated from a person’s weight and height that gives a rough indication of whether their body size falls into categories such as underweight, normal, overweight, or obese. For investors, BMI matters because it’s a common screening measure used in public health, insurance underwriting, clinical trials and market research; shifts in population BMI can affect demand for medical services, drugs, insurance costs and related business risks, like a quick fuel-gauge that signals broader health trends.

phase 3 medical

"based on -18.4% placebo-adjusted BMI reduction achieved by setmelanotide in global Phase 3 TRANSCEND trial"

Phase 3 is the late-stage clinical testing step for a new drug or medical treatment, where the product is given to large groups of patients to confirm effectiveness, monitor side effects, and compare it to standard care. Successful Phase 3 results are often the final scientific hurdle before regulators decide on approval and market launch—like passing a final exam before graduation—and can sharply change a company's valuation and future revenue prospects.

adverse events medical

"The most common adverse events (affecting >20% of participants) were skin..."

Adverse events are any harmful or unwanted medical occurrences experienced by people using a drug, device, or undergoing a treatment, whether or not the problem is caused by the product. Think of them as complaints or breakdowns noticed during a trial or after a product is on the market; regulators record and investigate them. Investors care because clusters or serious adverse events can delay approvals, trigger costly studies or recalls, change labeling, and quickly alter a company’s revenue and risk profile.

hyperpigmentation medical

"The most common adverse events (affecting >20% of participants) were skin hyperpigmentation, nausea..."

Hyperpigmentation is the darkening of patches of skin caused by extra pigment accumulating in certain areas, much like a coffee stain forming on fabric. It matters to investors because it is a common, visible condition that creates steady demand for prescription drugs, over‑the‑counter creams, cosmetic procedures and related diagnostics; trends in prevalence, new treatments or regulatory changes can directly affect revenue and market risk for companies in dermatology and beauty.

pediatric medical

"in adults and pediatric patients aged 4 years and older with acquired HO."

Relating to the medical care, products, or research specifically for infants, children, and adolescents. It matters to investors because treatments, devices, or drugs designed for this age group face different safety standards, testing requirements and market dynamics than adult products—think of it like tailoring clothing to fit a child rather than an adult, which changes design, approval hurdles and potential customer size, affecting regulatory risk and revenue prospects.

leptin receptor (LEPR) medical

"due to Bardet-Biedl syndrome (BBS) or Pro-opiomelanocortin (POMC), proprotein convertase subtilisin/kexin type 1 (PCSK1), or leptin receptor (LEPR) deficiency."

A leptin receptor (LEPR) is a protein on the surface of certain cells that detects the hormone leptin, which helps control appetite, energy use and body weight. For investors, LEPR is important because drugs, diagnostics or gene therapies that change how this “door” responds to leptin can affect treatment of obesity, diabetes and related conditions, so progress in research, patents or clinical trials around LEPR can materially influence a company's value.

monogenic obesity medical

"adult and pediatric patients aged 2 years and older with syndromic or monogenic obesity due to Bardet-Biedl syndrome..."

Monogenic obesity is excess weight caused primarily by a single change in one gene that disrupts how the body controls appetite, energy use or fat storage — like one broken switch in a complex control panel. It matters to investors because it creates clear targets for drugs and genetic tests, increasing the potential for precise therapies, faster clinical trials and defined patient groups that can make treatments more commercially viable.

AI-generated analysis. Not financial advice.

03/19/2026 - 06:30 PM

-- First and only FDA-approved therapy for the treatment of acquired hypothalamic obesity, a rare disease characterized by accelerated and sustained weight gain caused by hypothalamic injury or dysfunction –

-- Indicated to reduce excess body weight and maintain reduction long term in adults and pediatric patients aged 4 years and older with acquired hypothalamic obesity --

-- Approval based on -18.4% placebo-adjusted BMI reduction achieved by setmelanotide in global Phase 3 TRANSCEND trial [N=142] --

-- Company to host conference call today at 7:00 p.m. ET --

BOSTON, March 19, 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 U.S. Food and Drug Administration (FDA) has approved an expanded indication for IMCIVREE® (setmelanotide) to treat patients living with acquired hypothalamic obesity (HO).

Acquired HO is a rare disease characterized by accelerated and sustained weight gain caused by an injury to the hypothalamus or hypothalamic dysfunction. With this label expansion, IMCIVREE is indicated to reduce excess body weight and maintain reduction long term in adults and pediatric patients 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,” said David Meeker, M.D., Chairman, Chief Executive 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 responsible for controlling physiological functions such as energy expenditure, hunger, and weight regulation. Acquired HO most frequently follows tumors and their treatment or other hypothalamic injury or dysfunction. Based on analysis of the literature, tumor registries and claims data, Rhythm estimates there are approximately 10,000 people living with acquired HO in the U.S.

“Having a therapy for individuals and families affected by acquired hypothalamic obesity has the potential to be transformational,” said Amy Wood, Executive Director and Founder of the Raymond A. Wood Foundation. “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 positive pivotal Phase 3 TRANSCEND trial of setmelanotide in 142 patients with acquired HO. The global study met its primary endpoint, with a statistically significant -18.4% placebo-adjusted reduction in body mass index (BMI). For the primary endpoint of mean BMI change from baseline, study participants on setmelanotide therapy (n=94) achieved a -15.8% reduction compared with a +2.6% increase among patients on placebo (n=48) at 52 weeks (p<0.0001). Setmelanotide was generally well tolerated in the Phase 3 trial. The most common adverse events (affecting >20% of participants) were skin hyperpigmentation, nausea, vomiting and headache.

“Setmelanotide has shown effectiveness in targeting the underlying biology of acquired HO,” said Ashley Shoemaker, M.D., MSCI, Associate Professor of Pediatrics, Pediatric Endocrinology at Vanderbilt Health. “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) is also approved in the U.S. and Europe 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.

Rhythm is committed to supporting access for patients to its medicines and IMCIVREE® (setmelanotide) will be available for patients in the U.S. immediately. Rhythm InTune provides personalized, ongoing educational support for individuals living with certain rare forms of obesity. The program is designed for patients seeking education for themselves and their health care providers, assistance with insurance navigation at treatment initiation, injection support, and guidance on what to expect throughout treatment. For more information, contact patientsupport@rhythmtx.com.

Conference Call Information
Rhythm will host a live conference call and webcast at 7:00 p.m. ET today to discuss the FDA approval of IMCIVREE for patients with acquired HO. To access the live conference call, participants may register here.

A live webcast of the event will be available under "Events and Presentations" in the Investor Relations section of the Rhythm Pharmaceuticals website at https://ir.rhythmtx.com/. The archived webcast will be available on Rhythm Pharmaceuticals’ website approximately two hours after the event and will be available for 30 days following the event.

About Acquired Hypothalamic Obesity
Acquired hypothalamic obesity is a rare disease characterized by accelerated and sustained weight gain caused by an injury to the hypothalamus. Hypothalamic injury may lead to decreased alpha-melanocyte-stimulating hormone (α-MSH) production and impairment of MC4R pathway signaling. The MC4R pathway is responsible for regulating energy balance and body weight. Acquired hypothalamic obesity most frequently follows the growth or treatment of craniopharyngioma, astrocytoma or other hypothalamic-pituitary tumors. Additional causes of injury may include traumatic brain injury, stroke or inflammation. Due to impairment of the MC4R pathway, patients experience accelerated and sustained weight gain, often accompanied by hyperphagia and/or decreased energy expenditure. Acquired hypothalamic obesity can occur as early as six months following hypothalamic injury. Rhythm estimates there are approximately 10,000 people living with acquired HO in the U.S.

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 reduction long term in adults 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 & Healthcare Products Regulatory Agency (MHRA) have authorized setmelanotide 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. In the European Union and the United Kingdom, setmelanotide should be prescribed and supervised by a physician with expertise in obesity with underlying genetic etiology.

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 5% of IMCIVREE-treated patients and no placebo-treated patients. In patients with secondary adrenal insufficiency, monitor for clinical signs of acute adrenal insufficiency.

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 6% of IMCIVREE-treated patients and 2% of placebo-treated patients and hypernatremia in 5% of IMCIVREE-treated patients and 4% of placebo-treated patients. Monitor serum sodium levels with changes in fluid intake and hydration status. Adjust the doses of concomitant therapies for DI/AVP deficiency as needed.

ADVERSE REACTIONS
Most common adverse reactions (incidence ≥20% in at least 1 indication) included skin hyperpigmentation, injection site reactions, nausea, headache, diarrhea, abdominal pain, vomiting, depression, and spontaneous penile erection.

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, contact Rhythm Pharmaceuticals at +1 (833) 789-6337 or FDA at 1-800-FDA-1088 or http://www.fda.gov/medwatch. See section 4.8 of the Summary of Product Characteristics for information on reporting suspected adverse reactions in Europe.

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; the approval and use of IMCIVREE in patients with acquired hypothalamic obesity and its availability for patients; the commercial growth of IMCIVREE; our expectations surrounding potential regulatory submissions, progress, or approvals and timing thereof for any of our product candidates; the estimated market size and addressable population for our drug products, including IMCIVREE for the treatment of hypothalamic obesity in the United States, the European Union and Japan; the future announcement of data from our ongoing clinical trials; the ongoing enrollment in and potential progress or outcomes of our clinical trials; the presentation of the full data from the TRANSCEND study at an upcoming medical meeting; and the content, date and 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 Annual Report on Form 10-K for the year ended December 31, 2025 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 press release or to update them to reflect events or circumstances occurring after the date of this press release, whether as a result of new information, future developments or otherwise.

Corporate Contact:
David Connolly
Head of Investor Relations and Corporate Communications
Rhythm Pharmaceuticals, Inc.
dconnolly@rhythmtx.com

Media Contact:
Layne Cosgrove
Real Chemistry
llitsinger@realchemistry.com

Photos accompanying this announcement are available at:

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FAQ

What did Rhythm Pharmaceuticals (RYTM) announce on March 19, 2026 about IMCIVREE?

IMCIVREE received FDA approval for acquired hypothalamic obesity in patients aged 4 years and older. According to the company, approval was supported by the Phase 3 TRANSCEND trial showing a placebo-adjusted -18.4% BMI reduction.

How effective was setmelanotide in the Phase 3 TRANSCEND trial supporting RYTM's FDA approval?

Setmelanotide produced a placebo-adjusted -18.4% reduction in BMI at 52 weeks. According to the company, treated patients had -15.8% mean BMI change versus +2.6% increase on placebo (p<0.0001).

Who is eligible for IMCIVREE under the new RYTM FDA indication and when will it be available?

IMCIVREE is indicated for adults and pediatric patients aged 4 years and older with acquired hypothalamic obesity. According to the company, IMCIVREE will be available for U.S. patients immediately with Rhythm InTune support.

What safety issues were observed in the TRANSCEND trial for setmelanotide (RYTM)?

The most common adverse events (>20%) were skin hyperpigmentation, nausea, vomiting and headache. According to the company, setmelanotide was generally well tolerated in the Phase 3 trial.

How many people in the U.S. does Rhythm estimate are affected by acquired hypothalamic obesity?

Rhythm estimates approximately 10,000 people in the U.S. live with acquired hypothalamic obesity. According to the company, this estimate is based on literature, tumor registries and claims data analysis.

Rhythm Pharmaceuticals Announces FDA Approval of IMCIVREE® (setmelanotide) for Patients with Acquired Hypothalamic Obesity