Rhythm Pharmaceuticals, Inc. (Nasdaq: RYTM), a global
commercial-stage biopharmaceutical company focused on transforming
the lives of patients and their families living with rare
neuroendocrine diseases, today announced that the European
Commission (EC) expanded the marketing authorization for IMCIVREE®
(setmelanotide) to include children between 2 and younger than 6
years old with obesity due to Bardet Biedl syndrome (BBS) or
pro-opiomelanocortin (POMC), proprotein convertase subtilisin/kexin
type 1 (PCSK1), or leptin receptor (LEPR) deficiency.
“Today marks what we believe to be an important milestone for
European patients with BBS and POMC/LEPR deficiencies ages 2 to 6,
who now have the opportunity to address the severe and insatiable
hunger and early onset obesity associated with their conditions at
an early age,” said Yann Mazabraud, Executive Vice President, Head
of International at Rhythm Pharmaceuticals. “Early-onset obesity
that goes untreated can manifest in multiple comorbidities and
negatively affect quality of life and life expectancy. For the
first time, physicians and patients in Europe may have the
opportunity to access what we believe is a meaningful treatment
option for children as young as 2 years old.”
IMCIVREE is the first-ever authorized precision medicine in the
European Union for control of hunger and treatment of obesity in
adults and children, now as young as 2 years old, living with BBS
or POMC, PCSK1, or LEPR deficiency.
About Rhythm PharmaceuticalsRhythm 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) for chronic weight management in
adult and pediatric patients 6 years of age and older with
monogenic or syndromic obesity due to pro-opiomelanocortin (POMC),
proprotein convertase subtilisin/kexin type 1 (PCSK1) or leptin
receptor (LEPR) deficiency confirmed by genetic testing, or
patients with a clinical diagnosis of Bardet-Biedl syndrome (BBS).
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 6 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 IndicationIn the European
Union, 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 Europe, setmelanotide should be prescribed and
supervised by a physician with expertise in obesity with underlying
genetic etiology.
In the United States, setmelanotide is indicated for
chronic weight management in adult and pediatric patients 6 years
of age and older with monogenic or syndromic obesity due to POMC,
PCSK1 or 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) or BBS.
Limitations of UseSetmelanotide 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 POMC, PCSK1 or LEPR
deficiency, or BBS, including obesity associated with other genetic
syndromes and general (polygenic) obesity.
ContraindicationPrior serious hypersensitivity
to setmelanotide or any of the excipients in IMCIVREE. Serious
hypersensitivity reactions (e.g., anaphylaxis) have been
reported.
WARNINGS AND PRECAUTIONS
Skin Pigmentation and Darkening of Pre-Existing
Nevi: Generalized increased skin pigmentation and
darkening of pre-existing nevi have occurred because of its
pharmacologic effect. Full body skin examinations prior to
initiation and periodically during treatment should be conducted to
monitor pre-existing and new pigmentary lesions.
Heart rate and blood pressure
monitoring: In Europe, heart rate and blood
pressure should be monitored as part of standard clinical practice
at each medical visit (at least every 6 months) for patients
treated with setmelanotide.
Disturbance in Sexual Arousal: Spontaneous
penile erections in males and sexual adverse reactions in females
have occurred. Patients who have an erection lasting longer than 4
hours should seek emergency medical attention.
Depression and Suicidal Ideation: Depression
and suicidal ideation have occurred. Patients should be monitored
for new onset or worsening depression or suicidal thoughts or
behaviors. Consideration should be given to discontinuing
setmelanotide 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 setmelanotide.
Pediatric Population: The prescribing
physician should periodically assess response to setmelanotide
therapy. In growing children, the impact of weight loss on growth
and maturation should be evaluated. In Europe, the prescribing
physician should monitor growth (height and weight) using age- and
sex-appropriate growth curves.
Risk of Serious Adverse Reactions Due to Benzyl Alcohol
Preservative in Neonates and Low Birth Weight
Infants: Setmelanotide 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 ≥20%) included skin
hyperpigmentation, injection site reactions, nausea, headache,
diarrhea, abdominal pain, vomiting, depression, and spontaneous
penile erection.
USE IN SPECIFIC POPULATIONS
Lactation: Not recommended when
breastfeeding.
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 StatementsThis 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 of any of
our products or product candidates, including setmelanotide; 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; our expectations surrounding potential regulatory
submissions, and approvals, including within the EU; our business
strategy and plans, including regarding commercialization of
setmelanotide in the EU and other international regions; 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, general
economic conditions, and the other important factors discussed
under the caption “Risk Factors” in our Quarterly Report on Form
10-Q for the three months ended March 31, 2024 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 Contact:David ConnollyHead of
Investor Relations and Corporate CommunicationsRhythm
Pharmaceuticals, Inc.857-264-4280dconnolly@rhythmtx.com
Media Contact:Adam DaleyBerry & Company
Public Relations212-253-8881adaley@berrypr.com
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