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 Italian Medicine
Agency (AIFA) approved reimbursement (Gazzetta Ufficiale) for
IMCIVREE® (setmelanotide) for the treatment of obesity and control
of hunger associated with Bardet-Biedl syndrome (BBS).
“We are pleased that AIFA has recognized the need for a
precision medicine for obesity and control of hunger in BBS,” said
Yann Mazabraud, Executive Vice President and Head of International
at Rhythm. “Now families in Italy who are affected by this
rare neuroendocrine disease will be able to access the first
and only treatment option authorized in the European Union, and we
look forward to working with the community in Italy to deliver
IMCIVREE to patients as we aim to expand access in Europe
country-by-country.”
BBS is a rare melanocortin-4 receptor (MC4R) pathway disease
with hallmark symptoms of hyperphagia, a pathological hunger that
leads to abnormal food-seeking behaviors, and early-onset, severe
obesity. BBS is also associated with cognitive impairment,
polydactyly, renal dysfunction, hypogonadism, and visual
impairment. In Italy, the Company estimates the prevalence of BBS
to be between 1/125,000 and 1/160,000, with approximately 200
identified.
AIFA previously approved reimbursement for IMCIVREE for the
treatment of obesity and control of hunger associated with
biallelic proopiomelanocortin (POMC), proprotein convertase
subtilisin/kexin type 1 (PCSK1), or biallelic leptin receptor
(LEPR) deficiency.
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 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 Bardet-Biedl syndrome (BBS) or genetically
confirmed loss-of-function biallelic proopiomelanocortin (POMC),
including PCSK1, deficiency or biallelic leptin receptor (LEPR)
deficiency in adults and children 6 years of age and above.
In Canada, setmelanotide is indicated for the treatment of
obesity due to Bardet-Biedl syndrome (BBS) or genetically-confirmed
biallelic pro-opiomelanocortin (POMC), proprotein convertase
subtilisin/kexin type 1 (PCSK1), or leptin receptor (LEPR)
deficiency due to variants interpreted as pathogenic, likely
pathogenic, or of uncertain significance 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-fetal 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
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 our business strategy and plans, including
regarding commercialization of IMCIVREE in Italy, the European
Union, and elsewhere. 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 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, 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 three months ended September 30,
2023 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
A PDF accompanying this announcement is available
at http://ml.globenewswire.com/Resource/Download/0351ffea-fdca-47d7-9a4d-f7f0b79e1bdd
Rhythm Pharmaceuticals (NASDAQ:RYTM)
過去 株価チャート
から 4 2024 まで 5 2024
Rhythm Pharmaceuticals (NASDAQ:RYTM)
過去 株価チャート
から 5 2023 まで 5 2024