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 French National Agency for
Medicines and Health Products Safety (ANSM) and French National
Authority for Health (HAS) have granted pre-marketing early access
authorization AP1 (Autorisation d’Accès Précoce), for
IMCIVREE® (setmelanotide), an MC4R agonist, for patients with
lesional hypothalamic obesity.
AP1 allows for early access to innovative
therapies in France prior to European regulatory approval when a
positive benefit/risk ratio is recognized and when no other
therapeutic alternatives are available. The AP1 for setmelanotide
was granted following review of efficacy and safety data from
Rhythm’s Phase 2 trial by the ANSM and HAS. Products included in
the AP1 programs are fully covered by France’s National Health
System and Rhythm can expect to be reimbursed for any patients
receiving treatments through this program.
Setmelanotide has been available through a
similar program for patients in France with Bardet-Biedl syndrome
(BBS) since July 2022.
“We are delighted to announce that the French
regulatory authorities granted this unique AP1 status to
setmelanotide, as this is a recognition of the seriousness of the
unmet medical need and what we believe to be a significant first
step towards our goal of expanding access to setmelanotide to
include patients with hypothalamic obesity globally,” said Yann
Mazabraud, Executive Vice President and Head of International at
Rhythm. “We look forward to continuing to work with French experts
to deliver setmelanotide to patients in France.”
Hypothalamic obesity is a rare, acquired 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 increase in hunger in the first six to 12
months following tumor resection, and ultimately develop severe
obesity. Rhythm estimates there are approximately 500-2,000
patients living with hypothalamic obesity in France, with
approximately 50 to 200 new cases each year. In the United States,
the Company estimates the prevalence in hypothalamic obesity to be
approximately 5,000-10,000 patients with an annual incidence of
approximately 500 new patients.
As previously announced, Rhythm’s Phase 3 trial in hypothalamic
obesity is ongoing following strong Phase 2 trial data that showed
meaningful weight loss was sustained and progressed in patients
with hypothalamic obesity treated with setmelanotide. The Company
enrolled 18 patients with hypothalamic obesity in its Phase 2
trial. Sixteen of 18 patients (89%) achieved the primary endpoint
of 5 percent or greater reduction in body mass index (BMI) after 16
weeks of treatment. Overall, there was a 14.5 mean percent
reduction in BMI (N=18) at Week 16 from baseline.
“There are currently no effective or approved therapeutic
options for patients with hypothalamic obesity, and it is important
to understand that efforts to control weight and appetite with
traditional lifestyle changes are not sufficient,” said Professor
Christine Poitou, Professor of Nutrition, Pitié-Salpêtrière
University Hospital, Paris. “This AP1 addresses a significant unmet
need for patients with severe obesity and hyperphagia, two hallmark
symptoms of hypothalamic obesity, for the first time.”
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) 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 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 UseSetmelanotide 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 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 Statements
This 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 for patients with hypothalamic
obesity, our expectations surrounding potential regulatory
submissions, approvals and timing thereof, and our business
strategy and plans, including regarding commercialization of
IMCIVREE in France, the United States and other international
regions, including expectations surrounding coverage and
availability of IMCIVREE in France and related revenues. 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, 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, 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 March 31, 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 ConnollyExecutive
Director, Investor Relations and Corporate CommunicationsRhythm
Pharmaceuticals, Inc.857-264-4280dconnolly@rhythmtx.com
Media Contact:Adam DaleyBerry & Company
Public Relations212-253-8881adaley@berrypr.com
Rhythm Pharmaceuticals (NASDAQ:RYTM)
Gráfico Histórico do Ativo
De Abr 2024 até Mai 2024
Rhythm Pharmaceuticals (NASDAQ:RYTM)
Gráfico Histórico do Ativo
De Mai 2023 até Mai 2024