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 that the European Medicines Agency (EMA)
has issued a positive opinion on the Company’s orphan drug
designation request for setmelanotide as a treatment for acquired
hypothalamic obesity.
“Acquired hypothalamic obesity is a serious disease with severe
implications for patients and families and no effective treatment
options,” said Yann Mazabraud, Executive Vice President and Head of
International at Rhythm. “We are pleased with this EMA positive
opinion as it underscores the significant unmet need for these
patients and the potential of setmelanotide to bring a precision
medicine approach that may benefit patients with this disease
across Europe.”
EMA orphan drug designation is granted to drugs intended for the
treatment, diagnosis, or prevention of life-threatening or
chronically debilitating conditions affecting no more than five in
10,000 individuals in the European Union.
Rhythm is evaluating setmelanotide in a global Phase 3 clinical
trial in acquired hypothalamic obesity and expects to complete
patient enrollment in the fourth quarter of 2023.
Acquired hypothalamic obesity is a rare form of extreme obesity
that occurs following damage to the hypothalamic region of the
brain, which includes the MC4R pathway and is responsible for
controlling physiological functions such as hunger and weight
regulation. It most frequently follows the growth or surgical
removal of craniopharyngioma, astrocytoma, or other rare brain
tumors. Patients experience rapid weight gain, a reduction in
energy expenditure, and an increase in hunger leading to severe
obesity within six to 12 months following tumor resection. Rhythm
estimates there are approximately 3,500 to 10,000 patients living
with acquired hypothalamic obesity in the European countries of
Germany, France, Spain, Italy, The Netherlands, and the United
Kingdom.1
About Rhythm Pharmaceuticals Rhythm 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) is approved by the U.S. Food and Drug
Administration (FDA) and authorized by the European
Commission (EC) and the UK’s Medicines & Healthcare
Products Regulatory Agency (MHRA) for use in accordance with
product labeling. 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 andi 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.
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 Use 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.
In Europe, Setmelanotide should be prescribed and
supervised by a physician with expertise in obesity with underlying
genetic etiology.
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 REACTIONS
The 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-feeding It 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.
Fertility No 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 of
setmelanotide and the potential benefits and expectations of
receipt of a positive opinion on the Company’s orphan drug
designation request for setmelanotide as a treatment for acquired
hypothalamic obesity; and expectations regarding the prevalence of
patients living with acquired hypothalamic obesity. 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, risks relating to our liquidity and
expenses, our ability to enroll patients in clinical trials, the
design and outcome of clinical trials, the ability to achieve
necessary regulatory approvals, risks associated with data analysis
and reporting, failure to identify and develop additional product
candidates, unfavorable pricing regulations, third-party
reimbursement practices or healthcare reform initiatives, risks
associated with the laws and regulations governing our
international operations and the costs of any related compliance
programs, our ability to obtain or maintain orphan drug
designations for setmelanotide or to obtain or maintain exclusivity
in any use, the impact of competition, risks relating to product
liability lawsuits, inability to maintain our collaborations, or
the failure of these collaborations, our reliance on third parties,
risks relating to intellectual property, our ability to hire and
retain necessary personnel, 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 the other important factors
discussed under the caption “Risk Factors” in our Quarterly Report
on Form 10-Q for the three months ended June 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 Connolly Executive
Director, Investor Relations and Corporate Communications Rhythm
Pharmaceuticals, Inc. 857-264-4280 dconnolly@rhythmtx.com
Media Contact: Adam Daley Berry & Company
Public Relations 212-253-8881 adaley@berrypr.com
1 Rhythm’s European prevalence estimate for hypothalamic obesity
is limited to the EU4, UK and the Netherlands. The total 2020
population estimates for the six key countries (Germany, France,
Spain, Italy, Netherlands, and UK) of 339,295,304 was used to reach
a final prevalence of 0.1-0.3 in 10,000 patients.
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