Rhythm Pharmaceuticals, Inc. (Nasdaq: RYTM), a commercial-stage
biopharmaceutical company focused on transforming the lives of
patients and their families living with hyperphagia and severe
obesity caused by rare melanocortin-4 receptor (MC4R) pathway
diseases, today announced the commercial launch of IMCIVREE®
(setmelanotide) in Germany for the treatment of obesity and control
of hunger associated with Bardet-Biedl syndrome (BBS).
The German Federal Joint Committee (G-BA) ruled that IMCIVREE is
eligible for reimbursement by Statutory Health Insurances for BBS.
The G-BA unanimously voted to exclude IMCIVREE from its lifestyle
exemption list for patients with BBS, as done previously for
biallelic pro-opiomelanocortin (POMC), including proprotein
convertase subtilisin/kexin type 1 (PCSK1), deficiency or biallelic
leptin receptor (LEPR) deficiency.
“We are excited and well prepared to bring IMCIVREE to patients
in Germany, which is an important market with many identified
patients in need of a therapy for hyperphagia and severe obesity
that significantly burden people living with BBS and their
families,” said Yann Mazabraud, Executive Vice President, Head of
International at Rhythm. “Receiving exemption status from the G-BA
to provide access to IMCIVREE is an important recognition of the
severity of BBS and further reinforces the critical distinction
between rare MC4R pathway diseases and general obesity. IMCIVREE is
the first and only precision medicine for patients with monogenic
and syndromic obesity.”
BBS is a rare genetic disease that affects between 4,000-5,000
people in the European Union (EU) and United Kingdom (UK), with an
estimated prevalence of 1,200 people in Germany. People living with
BBS may experience insatiable hunger, also known as hyperphagia,
and severe obesity beginning early in life. BBS may also be
associated with cognitive impairment, polydactyly, renal
dysfunction, hypogonadism, and visual impairment. IMCIVREE is the
first and only U.S. Food and Drug Administration (FDA)-approved and
European Commission (EC)-authorized therapy that targets a root
cause of hyperphagia and early-onset, severe obesity, and is the
only approved therapy for BBS in Germany.
“Until now, the insatiable hunger leading to significant obesity
has been a serious and unresolved medical issue for patients with
BBS, also leading to comorbidities and psychological stress. The
targeted therapy with setmelanotide could represent a turning point
for many patients and demonstrate the considerable potential of
precision medicine for patients with rare genetic forms of
obesity,” said Dr. Annette Grüters-Kieslich, emeritus professor and
scientist at the Institute for Experimental Pediatric Endocrinology
and Diabetology, Charité - Universitätsmedizin Berlin.
With this launch, IMCIVREE is now available to patients in nine
global markets, including the United States, for treatment of POMC,
PCSK1, or LEPR deficiency obesity and/or BBS. In 2022, Rhythm was
granted marketing authorization for IMCIVREE by the EC for BBS and
early-access authorization for setmelanotide in France. Rhythm
expects to launch IMCIVREE for the treatment of patients with BBS
in the Netherlands later this year and in Italy, Spain, and the UK
in 2024.
About Rhythm PharmaceuticalsRhythm is a
commercial-stage biopharmaceutical company committed to
transforming the lives of patients and their families living with
hyperphagia and severe obesity caused by rare melanocortin-4
receptor (MC4R) diseases. Rhythm’s lead asset, IMCIVREE
(setmelanotide), an MC4R agonist designed to treat hyperphagia and
severe obesity caused by rare MC4R pathway diseases, 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 MC4R
pathway diseases, as well as a preclinical suite of investigational
candidates 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 Bardet-Biedl syndrome (BBS) or genetically
confirmed loss-of-function biallelic pro-opiomelanocortin (POMC),
including PCSK1, deficiency or biallelic leptin receptor (LEPR)
deficiency in adults and children 6 years of age and above.
Limitations of UseIn the United States and
Europe, Setmelanotide should be prescribed and supervised by a
physician with expertise in obesity with underlying genetic
etiology.
Setmelanotide 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.
WARNINGS AND PRECAUTIONS
Skin Monitoring: Setmelanotide may lead to
generalized increased skin pigmentation and darkening of
pre-existing naevi because of its pharmacologic effect. Full body
skin examinations should be conducted annually to monitor
pre-existing and new skin pigmentary lesions before and during
treatment with setmelanotide.
Heart rate and blood pressure monitoring: 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.
Prolonged penile erection: Spontaneous penile
erections have been reported in clinical trials with setmelanotide.
Patients who have a penile erection lasting longer than 4 hours
should be instructed to seek emergency medical attention for
potential treatment of priapism.
Depression: In clinical trials, depression has
been reported in patients treated with setmelanotide. Patients with
depression should be monitored at each medical visit during
treatment with setmelanotide. Consideration should be given to
discontinuing setmelanotide if patients experience suicidal
thoughts or behaviors.
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. The prescribing physician should
monitor growth (height and weight) using age- and sex-appropriate
growth curves.
Excipients: This medicinal product contains 10
mg benzyl alcohol in each ml. Benzyl alcohol may cause allergic
reactions. Patients who are pregnant or breastfeeding should be
advised of the potential risk from the excipient benzyl alcohol,
which might accumulate over time and cause metabolic acidosis. This
medicinal product should be used with caution in patients with
hepatic or renal impairment, because of the potential risk from the
excipient benzyl alcohol which might accumulate over time and cause
metabolic acidosis.
Sodium: This medicinal product contains less
than 1 mmol sodium (23 mg) per dose, that is to say essentially
“sodium-free.”
ADVERSE REACTIONSThe most frequent adverse
reactions are hyperpigmentation (51%), injection site reaction
(39%), nausea (33%), and headache (26%).
USE IN SPECIFIC POPULATIONS
PregnancyThere are no data from the use of
setmelanotide in pregnant women. Animal studies do not indicate
direct harmful effects with respect to reproductive toxicity.
However, administration of setmelanotide to pregnant rabbits
resulted in decreased maternal food consumption leading to
embryo-foetal effects. As a precautionary measure, setmelanotide
should not be started during pregnancy or while attempting to get
pregnant as weight loss during pregnancy may result in fetal harm.
If a patient who is taking setmelanotide has reached a stable
weight and becomes pregnant, consideration should be given to
maintaining setmelanotide treatment as there was no proof of
teratogenicity in the nonclinical data. If a patient who is taking
setmelanotide and still losing weight gets pregnant, setmelanotide
should either be discontinued, or the dose reduced while monitoring
for the recommended weight gain during pregnancy. The treating
physician should carefully monitor weight during pregnancy in a
patient taking setmelanotide.
Breast-feedingIt is unknown whether
setmelanotide is excreted in human milk. A nonclinical study showed
that setmelanotide is excreted in the milk of nursing rats. No
quantifiable setmelanotide concentrations were detected in plasma
from nursing pups. A risk to the newborn/infant cannot be excluded.
A decision must be made whether to discontinue breastfeeding or to
discontinue/abstain from setmelanotide therapy taking into account
the benefit of breastfeeding for the child and the benefit of
therapy for the mother.
FertilityNo human data on the effect of
setmelanotide on fertility are available. Animal studies did not
indicate harmful effects with respect to fertility.
To report SUSPECTED ADVERSE REACTIONS, contact Rhythm
Pharmaceuticals at +1 (833) 789-6337. See Summary of Product
Characteristics’ APPENDIX V for a list of European national
reporting systems to communicate adverse reactions.
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 U.S.
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, the potential benefits of setmelanotide for patients
with BBS, and our expectations surrounding potential regulatory
submissions, approvals and timing thereof, and our business
strategy and plans, including regarding commercialization of
setmelanotide in Germany other international regions. 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 and general economic conditions 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 Annual Report on Form 10-K for the quarter ended
December 31, 2022 and our other filings with the U.S. 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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