Rhythm Pharmaceuticals Announces FDA Approval of IMCIVREE® (setmelanotide) for Patients as Young as 2 Years Old
Rhythm Pharmaceuticals (NASDAQ: RYTM) announced FDA approval for expanded indication of IMCIVREE® (setmelanotide) to treat children as young as 2 years old with syndromic or monogenic obesity due to Bardet-Biedl syndrome (BBS) or genetically confirmed POMC, PCSK1, and LEPR deficiencies. IMCIVREE is the first precision medicine targeting MC4R pathway impairment, addressing hyperphagia and obesity in these rare conditions.
The approval follows successful clinical trials showing significant weight and hunger reduction. The drug was previously approved for patients 6 years and older, and has received marketing authorization in the UK and EU for patients as young as 2 years. BBS affects approximately 4,000-5,000 people in the US, while POMC, PCSK1, and LEPR deficiency obesities affect 600-2,500 people.
Rhythm Pharmaceuticals (NASDAQ: RYTM) ha annunciato l'approvazione della FDA per l'indicazione ampliata di IMCIVREE® (setmelanotide) per trattare bambini a partire dai 2 anni con obesità sindromica o monogenica dovuta alla sindrome di Bardet-Biedl (BBS) o con carenze genetiche confermate di POMC, PCSK1 e LEPR. IMCIVREE è il primo farmaco di precisione che mira a ripristinare il funzionamento della via MC4R, affrontando l'iperfagia e l'obesità in queste condizioni rare.
L'approvazione segue a trial clinici di successo che hanno mostrato significative riduzioni del peso e della fame. Il farmaco era stato precedentemente approvato per pazienti di 6 anni e più ed ha ricevuto l'autorizzazione per la commercializzazione nel Regno Unito e nell'UE per pazienti a partire dai 2 anni. La sindrome di Bardet-Biedl colpisce circa 4.000-5.000 persone negli Stati Uniti, mentre le obesità da carenza di POMC, PCSK1 e LEPR interessano da 600 a 2.500 persone.
Rhythm Pharmaceuticals (NASDAQ: RYTM) anunció la aprobación de la FDA para la indicación ampliada de IMCIVREE® (setmelanotide) para tratar a niños tan jóvenes como 2 años con obesidad sindrómica o monogénica debido al síndrome de Bardet-Biedl (BBS) o con deficiencias genéticas confirmadas de POMC, PCSK1 y LEPR. IMCIVREE es el primer medicamento de precisión que apunta a la alteración de la vía MC4R, abordando la hiperfagia y la obesidad en estas raras condiciones.
La aprobación sigue a ensayos clínicos exitosos que mostraron una reducción significativa del peso y el hambre. El medicamento ya había sido aprobado para pacientes de 6 años o más, y ha recibido autorización de comercialización en el Reino Unido y la UE para pacientes de tan solo 2 años. El BBS afecta aproximadamente a 4,000-5,000 personas en EE. UU., mientras que las obesidades por deficiencia de POMC, PCSK1 y LEPR afectan a 600-2,500 personas.
리듬 제약 (NASDAQ: RYTM)은 베르데트-비들 증후군(BBS) 또는 유전적으로 확인된 POMC, PCSK1, LEPR 결핍으로 인해 비만이 있는 2세 이상의 어린이를 치료하기 위해 IMCIVREE® (setmelanotide)의 확대 적응증에 대한 FDA 승인을 발표했습니다. IMCIVREE는 MC4R 경로 손상을 목표로 하는 첫 번째 정밀 의약품으로, 이러한 희귀 질환에서의 과식증과 비만을 해결합니다.
이번 승인은 체중 및 식욕 감소가 유의미하게 나타난 성공적인 임상 시험에 이어 이루어졌습니다. 이 약물은 이전에 6세 이상의 환자를 위해 승인되었으며, 영국 및 EU에서 2세 이상의 환자를 위해 판매 허가를 받았습니다. BBS는 미국에서 약 4,000-5,000명에게 영향을 미치며, POMC, PCSK1 및 LEPR 결핍으로 인한 비만은 600-2,500명에게 영향을 줍니다.
Rhythm Pharmaceuticals (NASDAQ: RYTM) a annoncé l'approbation de la FDA pour l'indication élargie de IMCIVREE® (setmelanotide) afin de traiter les enfants dès l'âge de 2 ans atteints d'obésité syndromique ou monogénique due au syndrome de Bardet-Biedl (BBS) ou à des carences confirmées génétiquement de POMC, PCSK1 et LEPR. IMCIVREE est le premier médicament de précision ciblant l'altération de la voie MC4R, s'attaquant à l'hyperphagie et à l'obésité dans ces conditions rares.
Cette approbation fait suite à des essais cliniques réussis montrant une réduction significative du poids et de la faim. Le médicament avait été précédemment approuvé pour les patients de 6 ans et plus, et a reçu une autorisation de mise sur le marché au Royaume-Uni et dans l'UE pour les patients dès l'âge de 2 ans. Le BBS touche environ 4 000 à 5 000 personnes aux États-Unis, tandis que les obésités dues aux carences de POMC, PCSK1 et LEPR touchent entre 600 et 2 500 personnes.
Rhythm Pharmaceuticals (NASDAQ: RYTM) hat die FDA-Zulassung für eine erweiterte Indikation von IMCIVREE® (setmelanotide) angekündigt, um Kinder ab 2 Jahren mit syndromaler oder monogener Adipositas aufgrund des Bardet-Biedl-Syndroms (BBS) oder genetisch bestätigten Mangelzuständen von POMC, PCSK1 und LEPR zu behandeln. IMCIVREE ist das erste Präzisionsmedikament, das auf eine Beeinträchtigung des MC4R-Pathways abzielt und Hyperphagie sowie Fettleibigkeit bei diesen seltenen Erkrankungen behandelt.
Die Genehmigung folgt auf erfolgreiche klinische Studien, die eine signifikante Gewichts- und Hungerreduktion zeigten. Das Medikament wurde zuvor für Patienten ab 6 Jahren zugelassen und hat in Großbritannien und der EU die Marketinggenehmigung für Patienten ab 2 Jahren erhalten. BBS betrifft etwa 4.000-5.000 Menschen in den USA, während Adipositas durch Mängel an POMC, PCSK1 und LEPR 600-2.500 Menschen betrifft.
- Expanded market opportunity with indication for younger patients (age 2+)
- First-mover advantage as only precision medicine for these conditions
- Strong clinical trial results showing significant weight/hunger reduction
- Established presence in multiple major markets (US, UK, EU)
- Addressing rare diseases with competition (4,600-7,500 total US patients)
- patient population size may restrict revenue potential
- Common adverse events including skin hyperpigmentation and injection site reactions
- Complex administration as injectable medication for young children
Insights
In simpler terms: Imagine a car with a broken fuel gauge always showing empty - that's how these children's brains perceive hunger. IMCIVREE fixes this broken gauge, helping prevent severe obesity from an early age.
For investors: Think of this as extending a subscription service to a younger age group - it means longer customer lifetime value and potentially higher revenue per patient, which could positively impact RYTM's long-term revenue growth trajectory.
BOSTON, Dec. 20, 2024 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (Nasdaq: RYTM), a 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 include children as young as 2 years old. IMCIVREE is indicated to reduce excess body weight and maintain weight reduction long-term in 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.
BBS and POMC, PCSK1 and LEPR deficiencies are rare melanocortin-4 receptor (MC4R) pathway diseases with hallmark characteristics that include hyperphagia, or pathological, insatiable hunger and impaired satiety accompanied by persistent and abnormal food-seeking behaviors, and early-onset obesity. IMCIVREE is the first and only precision medicine to target impairment of the hypothalamic MC4R pathway, a root cause of hyperphagia and obesity due to BBS and POMC, PCSK1 and LEPR deficiencies in adults and children as young as 2 years old in the United States, as well as Europe.
“It’s important to understand that rare MC4R pathway diseases differ from general obesity as the insatiable hunger these patients experience is pathologic and a result of impairment to a pathway in the brain. With this insatiable hunger, most patients develop early-onset obesity before the age of 5,” said Ilene Fennoy, MD, MPH, pediatric endocrinologist, obesity specialist and professor of Pediatrics at Columbia University Medical Center. “Obesity in childhood, if untreated, can lead to a greater risk of severe and long-term health complications, making early intervention to treat obesity critical. With this expanded indication for IMCIVREE, patients now can receive a much needed, targeted treatment that we believe can address a root cause of their obesity at a very young age.”
Results from clinical trials demonstrate that setmelanotide delivers significant and sustained reductions in measures of weight and hunger. Results from the Phase 3 VENTURE trial were published in the peer-reviewed journal The Lancet Diabetes & Endocrinology in November 2024. The most common adverse events are skin hyperpigmentation, injection site reactions, diarrhea, nausea and headache.
“Today’s approval is welcome news for the BBS community and others with rare MC4R diseases who struggle with hyperphagia,” said Tim Ogden, President of the Bardet Biedl Syndrome Foundation. “Many children with BBS feel hungry or think about food regardless of how much or how recently they've eaten, leaving families to deal with children sneaking or stealing food, which makes daily life extremely stressful. Parents have enough to worry about and manage when their child has a multi-systemic syndrome like BBS and IMCIVREE can be an important tool for their obesity."
“Rhythm is focused on ensuring patients with these rare genetic diseases that are present at birth and manifest early in life have access to IMCIVREE as soon as possible,” said David Meeker, M.D., Chairman, Chief Executive Officer and President of Rhythm. “We remain steadfast in our commitment to continue rapidly advancing care and precision medicines that address the root cause of rare neuroendocrine diseases.”
IMCIVREE initially received approval from the FDA in November 2020 for patients 6 years old and older with POMC, PCSK1 or LEPR deficiencies and approval in June 2022 for use in patients with BBS. IMCIVREE has also received marketing authorization from the United Kingdom’s Medicines & Healthcare products Regulatory Agency (MHRA) and the European Commission (EC) for use in patients as young as 2 years of age.
BBS, which is diagnosed clinically, affects approximately 4,000 to 5,000 people in the U.S. People living with BBS may experience insatiable hunger, also known as hyperphagia, and severe obesity beginning early in life. BBS also may be associated with cognitive impairment, polydactyly, renal dysfunction, hypogonadism, and visual impairment. POMC, PCSK1 and LEPR deficiency obesities, caused by biallelic variants in the POMC, PCSK1 or LEPR genes, affect approximately 600 to 2,500 people in the United States.
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 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 LB54640 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 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 as determined by an FDA-approved test 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 BBS or POMC, PCSK1, or LEPR deficiency, including obesity associated with other genetic syndromes and general (polygenic) obesity
Contraindication
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 in males and sexual adverse reactions in females 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, suicidal ideation and depressed mood 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, darkening of pre-existing nevi, development of new melanocytic nevi and increase in size of existing melanocytic nevi have occurred. Perform a full body skin examination prior to initiation and periodically during treatment to monitor pre-existing and new pigmented lesions.
Risk of Serious Adverse Reactions Due to Benzyl Alcohol Preservative in Neonates and Low Birth Weight Infants: IMCIVREE is not approved for use in neonates or infants. Serious and fatal adverse reactions including “gasping syndrome” can occur in neonates and low birth weight infants treated with benzyl alcohol preserved drugs.
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, contact Rhythm Pharmaceuticals at +1 (833) 789-6337 or FDA at 1-800-FDA-1088 or 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 potential, safety, efficacy, and regulatory and clinical progress, potential regulatory submissions, approvals and timing thereof of setmelanotide and other product candidates; the potential benefits of any of our products or product candidates for any specific disease indication, in any particular patient population, or at any dosage, including the potential benefits of setmelanotide for pediatric patients with BBS or POMC, PCSK1, or LEPR deficiency; expectations surrounding potential clinical trial results, regulatory submissions and approvals; our business strategy and plans, including regarding commercialization of setmelanotide in the United States; 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, 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 discussed under the caption “Risk Factors” in Rhythm’s Quarterly Report on Form 10-Q for the three months ended September 30, 2024 and 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 Contact:
David Connolly
Head of Investor Relations and Corporate Communications
Rhythm Pharmaceuticals, Inc.
857-264-4280
dconnolly@rhythmtx.com
Media Contact:
Sheryl Seapy
Real Chemistry
(949) 903-4750
sseapy@realchemistry.com
FAQ
What is the new age range for IMCIVREE (RYTM) treatment after FDA approval?
What conditions does IMCIVREE (RYTM) treat?
How many patients in the US could potentially benefit from IMCIVREE (RYTM)?
What are the main side effects of IMCIVREE (RYTM)?
In which regions is IMCIVREE (RYTM) currently approved?
