Rhythm Pharmaceuticals, Inc. (Nasdaq: RYTM), a commercial-stage
biopharmaceutical company committed to transforming the care of
people living with rare genetic diseases of obesity, today
announced that it has submitted its Type II variation application
to the European Medicines Agency (EMA) for IMCIVREE®
(setmelanotide) for the treatment of obesity and control of hunger
in adult and pediatric patients six years of age and older with
Bardet-Biedl syndrome (BBS) or Alström syndrome.
“This marks an important milestone toward our goal of delivering
IMCIVREE globally to patients with BBS and, ultimately, many other
rare genetic diseases of obesity,” said David Meeker, M.D.,
Chairman, President and Chief Executive Officer of Rhythm.
“IMCIVREE achieved clinically meaningful and statistically
significant results in our Phase 3 trial in BBS and provided
evidence of marked and sustained weight loss in patients with
Alström syndrome treated in our Phase 2 and 3 trials. Based on
these data, we believe IMCIVREE will be the first medicine to
effectively address the severe, early-onset obesity and hyperphagia
that characterize these diseases. We look forward to working
closely with regulatory authorities in the European Union to
deliver IMCIVREE to these additional populations.”
“The BBS community in the EU is particularly well established,
with approximately 1,500 patients diagnosed and being cared for at
academic centers,” said Yann Mazabraud, Executive Vice President,
Head of International of Rhythm. “Importantly, many of these
patients present with the severe obesity and hyperphagia that
treatment with IMCIVREE is designed to address. We are eager to
continue our targeted efforts to increase understanding of BBS and
the potential benefits of IMCIVREE and, if authorised, look forward
to bringing this treatment to market quickly as a key step toward
transforming the care of people living with rare genetic diseases
of obesity.”
The EMA submission is based on data from Rhythm’s pivotal Phase
3 clinical trial of setmelanotide in patients with BBS or Alström
syndrome. The EMA submission is based on data from Rhythm’s pivotal
Phase 3 clinical trial of setmelanotide in patients with BBS or
Alström syndrome. As previously reported, the study met its primary
endpoint and all key secondary endpoints, with statistically
significant and clinically meaningful reductions in weight and
hunger at 52 weeks on therapy. All patients who met the
primary endpoint defined as more than 10 percent weight loss had
BBS and none had Alström syndrome. However, data from this Phase 3
trial is supported by results from the Phase 2 trial, which suggest
that treatment with setmelanotide may result in decreased weight
and hunger in people living Alström syndrome. In addition,
data from a predefined exploratory endpoint showed that, in BBS and
Alström syndrome patients younger than 18 years old, setmelanotide
treatment was associated with clinically meaningful reductions in
BMI-Z scores. The BMI-Z score, or BMI standard deviation score,
represents the number of standard deviations from median BMI by
child age and sex.
About Bardet-Biedl and Alström SyndromesBBS and
Alström syndrome are ultra-rare genetic diseases that affect
multiple organ systems. Clinical features of BBS may include
cognitive impairment, polydactyly, renal dysfunction, hypogonadism,
and visual impairment. Clinical features of Alström syndrome may
include progressive visual and auditory impairment, insulin
resistance and Type 2 diabetes, hyperlipidemia, progressive kidney
dysfunction, cardiomyopathy, and short stature in adulthood.
Insatiable hunger, also known as hyperphagia, and severe obesity
beginning early in life is common in people living with either BBS
or Alström syndrome. Rhythm estimates that BBS affects
approximately 1,500 to 2,500 people and that Alström syndrome
affects approximately 500 people in the United States, with a
similar prevalence estimate in Europe. Currently, there are no
approved therapies targeting the MC4R pathway for reducing body
weight and hunger in BBS or Alström syndrome.
About Rhythm PharmaceuticalsRhythm is a
commercial-stage biopharmaceutical company committed to
transforming the treatment paradigm for people living with rare
genetic diseases of obesity. Rhythm’s precision medicine, IMCIVREE
(setmelanotide), was approved in November 2020 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 obesity due to POMC, PCSK1 or LEPR deficiency
confirmed by genetic testing and in July and September 2021,
respectively, by the European Commission (EC) and Great
Britain’s Medicines & Healthcare Products Regulatory
Agency (MHRA) for the treatment of obesity and the control of
hunger associated with genetically confirmed loss-of-function
biallelic POMC, including PCSK1, deficiency or biallelic LEPR
deficiency in adults and children 6 years of age and above.
IMCIVREE is the first-ever FDA-approved and EC- and MHRA-authorized
therapy for patients with these rare genetic diseases of obesity.
Rhythm is advancing a broad clinical development program for
setmelanotide in other rare genetic diseases of obesity, and is
leveraging the Rhythm Engine and the largest known obesity DNA
database -- now with approximately 37,500 sequencing samples -- to
improve the understanding, diagnosis and care of people living with
severe obesity due to certain genetic deficiencies. Rhythm’s
headquarters is in Boston, MA.
IMCIVREE®
(setmelanotide) IndicationIn the EU and Great
Britain, IMCIVREE is indicated for the treatment of obesity and the
control of hunger associated with genetically confirmed
loss-of-function biallelic POMC, including PCSK1, deficiency or
biallelic LEPR deficiency in adults and children 6 years of age and
above. IMCIVREE should be prescribed and supervised by a physician
with expertise in obesity with underlying genetic etiology.
In the United States, IMCIVREE is indicated for chronic
weight management in adult and pediatric patients 6 years of age
and older with obesity due to proopiomelanocortin (POMC),
proprotein convertase subtilisin/kexin type 1 (PCSK1), or leptin
receptor (LEPR) deficiency. The condition must be confirmed by
genetic testing demonstrating variants in POMC, PCSK1,
or LEPR genes that are interpreted as pathogenic, likely
pathogenic, or of uncertain significance (VUS).
Limitations of UseIMCIVREE is not indicated for
the treatment of patients with the following conditions as IMCIVREE
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, including obesity associated with other genetic
syndromes and general (polygenic) obesity.
Important Safety Information
WARNINGS AND PRECAUTIONS
Disturbance in Sexual Arousal: Sexual
adverse reactions may occur in patients treated with IMCIVREE.
Spontaneous penile erections in males and sexual adverse reactions
in females occurred in clinical studies with IMCIVREE. Instruct
patients who have an erection lasting longer than 4 hours to seek
emergency medical attention.
Depression and Suicidal Ideation: Some
drugs that target the central nervous system, such as IMCIVREE, may
cause depression or suicidal ideation. Monitor patients for new
onset or worsening of depression. Consider discontinuing IMCIVREE
if patients experience suicidal thoughts or behaviors.
Skin Pigmentation and Darkening of Pre-Existing
Nevi: IMCIVREE may cause generalized increased skin
pigmentation and darkening of pre-existing nevi due to its
pharmacologic effect. This effect is reversible upon
discontinuation of the drug. Perform a full body skin examination
prior to initiation and periodically during treatment with IMCIVREE
to monitor pre-existing and new skin pigmentary 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.
ADVERSE REACTIONS
- The most common adverse reactions (incidence ≥23%) were
injection site reactions, skin hyperpigmentation, nausea, headache,
diarrhea, abdominal pain, back pain, fatigue, vomiting, depression,
upper respiratory tract infection, and spontaneous penile
erection.
USE IN SPECIFIC POPULATIONSDiscontinue IMCIVREE
when pregnancy is recognized unless the benefits of therapy
outweigh the potential risks to the fetus.
Treatment with IMCIVREE is not recommended for use while
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 Full Prescribing Information and EU
SmPC for IMCIVREE.
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
setmelanotide, our expectations surrounding potential regulatory
submissions, approvals and timing thereof, our business strategy
and plans, including regarding commercialization of setmelanotide,
and our participation in upcoming events and presentations.
Statements using word 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 liquidity and expenses, the impact of the
COVID-19 pandemic on our business and operations, including our
preclinical studies, clinical trials and commercialization
prospects, 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 quarterly period ended June 30,
2021 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
Investor Contact:Hannah DeresiewiczStern
Investor Relations,
Inc.212-362-1200hannah.deresiewicz@sternir.com
Media Contact:Adam DaleyBerry & Company
Public Relations212-253-8881adaley@berrypr.com
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