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
Medicines Agency’s (EMA) Committee for Medicinal Products for Human
Use (CHMP) has adopted a positive opinion recommending the
marketing authorization for IMCIVREE® (setmelanotide) 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.
“We continue to work closely with health authorities throughout
the European Union to make IMCIVREE available to eligible patients
with uncontrolled hunger and early-onset, severe obesity associated
with these rare neuroendocrine diseases,” said Yann Mazabraud,
Executive Vice President, Head of International at Rhythm
Pharmaceuticals. “We believe this positive CHMP opinion for
pediatric use underscores the severe impact of the disruption of
the MC4R pathway and significant unmet medical need for young
children. It is important to diagnose patients with these diseases
early in life before the comorbidities of severe obesity take
hold.”
IMCIVREE is the first-ever authorized treatment option in the
European Union for control of hunger and treatment of obesity and
in adults and children 6 years of age and above living with BBS or
POMC, PCSK1, or LEPR deficiency.
The CHMP opinion on the marketing authorization to include
patients as young as 2 years old now will be reviewed by the
European Commission (EC), which has the authority to grant
marketing authorizations for medicinal products in the European
Union (EU). A final decision on the application to expand the
marketing authorization for setmelanotide is anticipated in the
second half of 2024.
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 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.
In 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 6 years of age and above. In Europe,
setmelanotide should be prescribed and supervised by a physician
with expertise in obesity with underlying genetic etiology.
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 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, and 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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