Rhythm Pharmaceuticals Receives Positive CADTH Reimbursement Recommendation for IMCIVREE® (setmelanotide)
02 Novembre 2023 - 1:00PM
Rhythm Pharmaceuticals, Inc. (Nasdaq: RYTM), a global
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 Canadian Agency for
Drugs and Technologies in Health (CADTH) has recommended that
IMCIVREE
® (setmelanotide) be reimbursed by
CADTH-participating public drug plans, with conditions, for weight
management in adult and pediatric patients six years of age and
older with obesity due to Bardet-Biedl syndrome (BBS).
“This positive CADTH recommendation will be welcome news for the
patients and families living with hyperphagia, which is a
pathological and insatiable hunger, and the severe obesity that is
often associated with BBS,” said Prof. Andrea M. Haqq, M.D.,
Department of Pediatrics, Faculty of Medicine & Dentistry,
University of Alberta. “This important recommendation further
recognizes the unmet needs of people with BBS who otherwise have no
targeted precision treatment for weight management.”
CADTH’s recommendation is based on results from Rhythm’s Phase 3
trial that demonstrated treatment with setmelanotide resulted in
meaningful clinical benefit in patients 6 years of age and older
with obesity due to BBS. Data showed that treatment with
setmelanotide for 52 weeks resulted in a clinically meaningful
reduction in weight-related parameters, such as total body weight
and body mass index (BMI), and the results were supported by the
exploratory comparative 14-week placebo-controlled outcomes.
Consistent with prior clinical experience, setmelanotide was
generally well tolerated. Treatment-emergent adverse events (TEAEs)
included mild injection site reactions and nausea.
BBS is a rare genetic disease with an estimated prevalence of 1
in 100,000 to 160,000 in the populations of North America and
Europe with an estimated 300-400 people living with the disease in
Canada. People living with BBS may experience insatiable hunger,
also known as hyperphagia, and severe obesity beginning early in
life. BBS may also be associated with cognitive impairment,
polydactyly, renal dysfunction, hypogonadism, and visual
impairment.
IMCIVREE® (setmelanotide) is the first and only therapy approved
in Canada for weight management in adult and pediatric patients 6
years of age and older with obesity due to BBS. IMCIVREE®
(setmelanotide) was given a Priority Review by Health Canada.
“This recommendation represents a significant milestone for
Canadian patients living with obesity due to BBS. We appreciate
CADTH’s recommendation and their recognition of the rare genetic
nature of BBS, the unmet needs of these patients and that IMCIVREE
is the only available therapy that targets the underlying disease
mechanism of BBS,” said Carol Stiff, General Manager of Rhythm
Pharmaceuticals Canada Inc.
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.
To report SUSPECTED ADVERSE REACTIONS, contact Rhythm
Pharmaceuticals at +1 (833) 789-6337. You can report any
suspected side effects associated with the use of health products
to Health Canada by: Visiting the Web page on Adverse Reaction
Reporting
(https://www.canada.ca/en/health-canada/services/drugs-health-products/medeffect-canada.html)
for information on how to report online, by mail or by fax; or
calling toll-free at 1-866-234-2345.
Please see the Product Monograph at
https://rhythmtx.ca/wp-content/uploads/2023/05/IMCIVREE-Product-Monograph-EN.pdf
for complete 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,
the potential benefits of and our expectations surrounding the
CADTH reimbursement recommendation, and our business strategy and
plans, including regarding commercialization of IMCIVREE in Canada
and other international regions. 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 DaleyBerry & Company
Public Relations212-253-8881adaley@berrypr.com
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