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 an Expanded Access Program (EAP) for setmelanotide for
the treatment of eligible patients in the United States with severe
obesity and hyperphagia due to Bardet-Biedl Syndrome (BBS). Rhythm
expects to submit to the U.S. Food and Drug Administration (FDA) a
supplemental new drug application (sNDA) for setmelanotide for the
treatment of adult and pediatric patients 6 years of age and older
with hunger and obesity due to BBS by the end of September.
“BBS is a serious disease with significant unmet needs,” said
David Meeker, M.D., Chair, President and Chief Executive Officer of
Rhythm. “Following the completion of our Phase 3 clinical trial and
as we advance through the regulatory review process, we are pleased
to make setmelanotide available through an expanded access program
to eligible patients in the United States who are living with BBS.
This program reflects our commitment to delivering therapies for
rare genetic diseases of obesity as rapidly as possible.”
The FDA’s expanded access regulations are designed to facilitate
access to an investigational therapy to treat patients who are
unable to participate in clinical trials and have serious or
immediately life-threatening diseases or conditions for which there
are no comparable or satisfactory alternative treatment
options.
If a treating physician believes setmelanotide may be the only
therapeutic option for a patient with severe obesity and
hyperphagia due to BBS, the physician can request additional
information by contacting ExpandedAccess@rhythmtx.com.
About Rhythm PharmaceuticalsRhythm is a
commercial-stage biopharmaceutical company committed to
transforming the treatment paradigm for people living with rare
genetic diseases of obesity. The Company’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 by
the European Commission (EC) in July 2021 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
and EC-approved 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. The Company 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. The company is based in Boston, MA.
IMCIVREE® (setmelanotide)
IndicationIn 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).
In the EU, 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.
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 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, and our business strategy and plans, including regarding
commercialization of setmelanotide. 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,
the impact of our management transition, 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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