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 accepted the company’s supplemental New Drug Application (sNDA)
for IMCIVREE® (setmelanotide), a melanocortin-4 receptor (MC4R)
agonist, for the treatment of obesity due to Bardet-Biedl syndrome
(BBS) or pro-opiomelanocortin (POMC), including proprotein
convertase subtilisin/kexin type 1 (PCSK1), or leptin receptor
(LEPR) deficiency obesity in children as young as 2 years old. The
FDA has granted Priority Review of the sNDA and assigned a
Prescription Drug User Fee Act (PDUFA) goal date of December 26,
2024.
“This milestone brings us another step closer to offering a
precision therapy for young patients in the U.S. experiencing
hyperphagia – a pathological hunger that leads to aberrant
food-seeking behaviors – and severe obesity caused by rare MC4R
pathway diseases,” said David Meeker, M.D., Chairman, Chief
Executive Officer and President of Rhythm. “IMCIVREE has been
approved for more than two years and prescribed to hundreds of
patients 6 years old and older, and we believe that treating
patients at an even earlier age will positively affect the lives of
these children and their families.”
The sNDA submission is based on data from Rhythm’s multi-center,
one-year, open-label Phase 3 trial in patients (N=12) between 2 and
younger than 6 years old with obesity due to biallelic POMC/PCSK1
or LEPR deficiency or a clinical diagnosis of BBS. The hyperphagia
and severe obesity of rare genetically-caused MC4R pathway diseases
can present early in life, and these data showed potential efficacy
in patients younger than 6 years of age. Treatment with
setmelanotide achieved the primary endpoint with a 3.04 mean
reduction in BMI-Z score (a measure of body mass index deviations
from what is considered normal) and 18.4 percent mean reduction in
BMI.
In July 2024, the European Commission (EC) expanded the
marketing authorization for IMCIVREE to include children as young
as 2 years old with obesity due to BBS or POMC/PCSK1, or LEPR
deficiency. In the United States, IMCIVREE is currently 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), and BBS.
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. The EC
has also authorized setmelanotide for control of hunger and
treatment of obesity in children as young as 2 years old, living
with BBS or POMC, PCSK1, or LEPR deficiency. 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 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.
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 U.S. Prescribing Information and EU
Summary of Product Characteristics 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,
including the PDUFA goal date of our sNDA discussed herein, the
potential benefits of any of the Company’s 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; expectations
surrounding potential regulatory submissions and approvals,
including within the United States, the EU and other regions;
business strategy and plans, including regarding commercialization
of setmelanotide in the United States, the EU and other 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 Rhythm’s Quarterly
Report on Form 10-Q for the three months ended June 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 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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