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 National Institute for Health
and Care Excellence (NICE) has issued guidance that recommends
IMCIVREE® (setmelanotide) as an option for treating obesity
and controlling hunger caused by pro-opiomelanocortin (POMC)
deficiency, including proprotein convertase subtilisin/kexin type 1
(PCSK1), or leptin receptor (LEPR) deficiency in people six years
of age and over. With this recommendation under the Highly
Specialised Technologies (HST) pathway, IMCIVREE will be funded and
available for use within 90 days in the National Health Service.
POMC and LEPR deficiencies are caused by genetic
variants that disrupt signaling of the MC4R pathway, a system in
the hypothalamus that regulates hunger, satiety and energy
expenditure. Disrupted MC4R signaling causes hyperphagia and
early-onset, severe obesity. People living with obesity due to POMC
or LEPR deficiency struggle with insatiable hunger, also known as
hyperphagia, and extreme obesity beginning at a young age.1,2 These
diseases severely affect the quality of life of people living with
them, as well as that of their families and caregivers, with many
reporting a significant psychological burden, which can manifest as
poor mental health, low self-esteem or depression. NICE concluded
that obesity caused by POMC or LEPR deficiency is a debilitating
condition associated with multiple comorbidities.
Alex Potter, a resident of England who participated
in the Rhythm clinical trial and served as a patient representative
in the NICE deliberations, said, “LEPR deficiency casts a shadow
over every aspect of life. Hunger permeates every thought, action,
and conversation. The continuous search for food and severe weight
gain has affected my engagement in normal daily activities, which
in turn makes building meaningful relationships challenging. For
me, participating in this trial has always been about giving other
people the chance to live a more normal life - one I never had.
This decision will affect the lives of so many families who, like
me, continue to be physically and psychologically impacted by this
disease.”
“The effects of rare MC4R pathway diseases,
including POMC and LEPR deficiency, go far beyond a patients’
weight and hunger, severely affecting their ability to maintain a
normal quality of life beginning in childhood,” said Sadaf Farooqi,
M.D., Ph.D., Professor at the Wellcome-MRC Institute of Metabolic
Science and NIHR Cambridge Biomedical Research Centre. “In
addition, caring for a person with one of these conditions can be
physically and mentally draining for families who are often
stigmatized in their communities. This NICE recommendation reflects
the value of IMCIVREE and it is welcome news for clinicians and
eligible patients who will soon have access to the first and only
therapy to address the underlying cause of obesity and hunger in
POMC and LEPR deficiencies.”
The final NICE recommendation is aligned to the
European Marketing Authorization (EMA) and UK Medicines and
Healthcare Products Regulatory Agency (MHRA) approval as the only
authorised treatment option for these rare genetic diseases of
obesity. The most common adverse events were skin
hyperpigmentation, injection site reactions, nausea and
headache.
“We are delighted by this positive NICE
recommendation, which makes available the first therapeutic option
for patients with POMC or LEPR deficiency in England and Wales,”
said David Meeker, M.D., Chair, President and Chief Executive
Officer of Rhythm. “We are grateful to NICE for their close
partnership throughout this review process and look forward to
continued collaboration with Health Technology Assessment bodies
and payers across Europe as we execute on our country-by-country
strategy to achieve market access for IMCIVREE.”
The full text of the NICE recommendation can be
found at:
https://www.nice.org.uk/guidance/hst21/chapter/1-Recommendations.
Intended for UK healthcare professionals.
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) pathway
diseases. Rhythm’s precision medicine, IMCIVREE (setmelanotide), 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 POMC, PCSK1 or
LEPR deficiency confirmed by genetic testing, or patients with a
clinical diagnosis of Bardet-Biedl syndrome (BBS). The European
Commission (EC) and Great Britain’s Medicines & Healthcare
Products Regulatory Agency (MHRA) have authorized IMCIVREE 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-approved and EC- and MHRA-authorized therapy for patients
living with these rare genetic diseases of obesity. The Company
submitted a Type II variation application to the European Medicines
Agency seeking regulatory approval and authorization for
setmelanotide to treat obesity and control of hunger in adult and
pediatric patients 6 years of age and older with BBS in the
European Union. Additionally, Rhythm is advancing a broad clinical
development program for setmelanotide in other rare genetic
diseases of obesity and is leveraging the Rhythm Engine and the
largest known obesity DNA database -- now with approximately 45,000
sequencing samples -- to improve the understanding, diagnosis and
care of people living with severe obesity due to certain genetic
deficiencies. Rhythm’s headquarters is in Boston, MA.
IMCIVREE®
(setmelanotide) IndicationIn Great Britain and the
European Union, IMCIVREE is indicated for the treatment of obesity
and the control of hunger associated with 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.
In the United States, IMCIVREE 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 Bardet-Biedl syndrome (BBS).
Limitations of UseIMCIVREE 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 generalised
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 IMCIVREE. Consideration should
be given to discontinuing IMCIVREE if patients experience suicidal
thoughts or behaviours.
Paediatric 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.
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, IMCIVREE 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 IMCIVREE 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.
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 our
expectations surrounding potential regulatory submissions,
approvals and timing thereof, and our business strategy and plans,
including regarding commercialization of IMCIVREE in the
United Kingdom, the United States and other international
regions, including expectations surrounding funding and
availability of IMCIVREE in the National Health Service and related
timing. 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, the ability to obtain or maintain
coverage and adequate reimbursement for IMCIVREE or our other
product candidates, 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 quarter ended March 31, 2022 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
Georgia Wellstedemotive
agency+44 020 8106 7891georgia.wellsted@emotiveagency.com
UK-SET-2200004
07/2022
1 Ayers KL, Glicksberg BS, Garfield AS, et al.
Melanocortin 4 receptor pathway dysfunction in obesity: patient
stratification aimed at MC4R agonist treatment. J Clin Endocrinol
Metab. 2018;103(7):2601-2612.2 Krude H, Biebermann H, Luck W, Horn
R, Brabant G, Grüters A. Severe early-onset obesity, adrenal
insufficiency and red hair pigmentation caused by POMC variants in
humans. Nat Genet. 1998;19(2):155-157.
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