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 Haute Autorité de
santé (HAS) have granted pre-marketing authorization AP1
(autorisation d’accès précoce - early access authorization), for
IMCIVREE® (setmelanotide), an MC4R agonist, for patients with
genetically-confirmed Bardet-Biedl syndrome (BBS) for the treatment
of obesity and the control of hunger.
AP1 allows for early access to innovative
therapies in France prior to 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 clinical studies
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.
“We are delighted to announce that the French
regulatory authorities granted AP1 status to setmelanotide, making
it available for patients with BBS living with hyperphagia and
severe obesity,” said Yann Mazabraud, Executive Vice President and
Head of International at Rhythm. “We look forward to collaborating
with the broad and established network of French BBS experts to
deliver setmelanotide to patients living with BBS in France, as we
work to transform the care of patients with BBS globally.”
BBS is a rare genetic disease that affects
approximately 1,500-2,500 people in the United States and
approximately 2,500 patients in Europe. In France, Rhythm estimates
that there are approximately 700 patients diagnosed with BBS. In
addition to insatiable hunger, known as hyperphagia, and severe
obesity, BBS is also associated with cognitive impairment,
polydactyly, renal dysfunction, hypogonadism, and visual
impairment. Impairments in the MC4R pathway are a root cause of the
early-onset obesity and hyperphagia associated with BBS.
“It is important to understand that efforts to
control weight and appetite with traditional lifestyle changes are
not effective for people who are suffering from the extreme hunger
and weight gain associated with rare MC4R pathway diseases,” said
Hélène Dolffus, M.D., Ph.D., Professor of Genetics at Hôpitaux
Universaires de Strasbourg, Head of the 1112 INSERM Research Unit
and Head of the European Rare Diseases Network - EYE. “This AP1
addresses a significant unmet need for patients with severe obesity
and hyperphagia, two hallmark symptoms of BBS.”
Rhythm’s Type II variation application to the
European Medicines Agency (EMA) for the treatment of obesity and
control of hunger in adult and pediatric patients 6 years of age
and older with BBS is under review. Rhythm anticipates that the
EMA’s Committee for Medicinal Products for Human Use (CHMP) will
make its recommendation on this application in the third quarter of
2022, with a final decision from the European Commission expected
to follow.
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 the EU and Great Britain, IMCIVREE is
indicated 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 should be
prescribed and supervised by a physician with expertise in obesity
with underlying genetic etiology.
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:
- Pro-opiomelanocortin (POMC), proprotein convertase
subtilisin/kexin type 1 (PCSK1) or leptin receptor (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)
- Bardet-Biedl syndrome (BBS)
Limitations of UseIMCIVREE is not indicated for
the treatment of patients with the following conditions as IMCIVREE
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
Disturbance in Sexual Arousal: Spontaneous
penile erections in males and sexual adverse reactions in females
have occurred. Inform patients that these events may occur and
instruct patients who have an erection lasting longer than 4 hours
to seek emergency medical attention.
Depression and Suicidal Ideation: Depression
and suicidal ideation have occurred. Monitor patients for new onset
or worsening depression or suicidal thoughts or behaviors. Consider
discontinuing IMCIVREE if patients experience suicidal thoughts or
behaviors, or clinically significant or persistent depression
symptoms occur.
Skin Pigmentation and Darkening of Pre-existing
Nevi: Generalized increased skin pigmentation and
darkening of pre-existing nevi have occurred. Perform a full body
skin examination prior to initiation and periodically during
treatment to monitor pre-existing and new pigmentary lesions.
Risk of Serious Adverse Reactions Due to Benzyl Alcohol
Preservative in Neonates and Low Birth Weight Infants:
IMCIVREE is not approved for use in neonates or infants. Serious
and fatal adverse reactions including “gasping syndrome” can occur
in neonates and low birth weight infants treated with benzyl
alcohol-preserved drugs.
ADVERSE REACTIONS
- The most common adverse reactions (incidence ≥20%) included
skin hyperpigmentation, injection site reactions, nausea, headache,
diarrhea, abdominal pain, vomiting, depression, and spontaneous
penile erection.
USE IN SPECIFIC POPULATIONSTreatment with
IMCIVREE is not recommended when breastfeeding. Discontinue
IMCIVREE when pregnancy is recognized unless the benefits of
therapy outweigh the potential risks to the fetus.
To report SUSPECTED ADVERSE REACTIONS, contact Rhythm
Pharmaceuticals at 833-789-6337 or FDA at 1-800-FDA-1088 or
www.fda.gov/medwatch.
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 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, 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
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