Rhythm Pharmaceuticals, Inc. (Nasdaq: RYTM), a commercial-stage
biopharmaceutical company, today announced that Health Canada has
approved IMCIVREE® (setmelanotide solution for subcutaneous
injection) for weight management in adult and pediatric patients 6
years of age and older with 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. Please
consult the IMCIVREE product monograph available at
www.rhythmtx.ca.
“We are pleased to announce Health Canada’s approval of
IMCIVREE, marking a significant expansion of our footprint in North
America and another important step forward in our efforts to
deliver our precision medicine to people worldwide,” said Jennifer
Chien, Executive Vice President, Head of North America for Rhythm.
“We will leverage insights from our experience with launches in the
U.S. and Europe to support the strong team we have in Canada and
look forward to making IMCIVREE commercially available in Canada in
the months ahead.”
The early-onset obesity in patients with BBS and POMC, PCSK1 and
LEPR deficiencies are caused by genetic variants that result in
impairments in the melanocortin-4 receptor (MC4R) pathway, a system
in the hypothalamus that regulates hunger, satiety, and energy
expenditure. IMCIVREE is the first and only therapy approved in
Canada for weight management in these patients following a Priority
Review, which shortened the review process and was granted based on
the serious nature of these conditions and lack of treatment
options in Canada.
IMCIVREE is not indicated for patients with obesity due to
suspected POMC, PCSK1, or LEPR deficiency with POMC, PCSK1, or LEPR
variants classified as benign or likely benign, or 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, as it would not be expected to be
effective.
“Research has shown that the burden of severe obesity negatively
affects the lives of patients and families,” said Prof. Andrea M.
Haqq, M.D., Department of Pediatrics, Faculty of Medicine &
Dentistry, University of Alberta. “Access to a therapy that
addresses an underlying cause of their obesity is a significant
advancement for patients and their families living with BBS and
POMC, PCSK1 and LEPR deficiencies.”
Jill Hamilton, M.D., Pediatric Endocrinologist at The Hospital
for Sick Children, added, “This approval will bring much-needed
relief to patients and their families who need a therapeutic option
that addresses an underlying cause of obesity associated with these
rare MC4R pathway diseases. Setmelanotide has been shown to reduce
the weight of patients living with BBS or POMC, PCSK1 or LEPR
deficiency. The Health Canada approval will provide access to many
patients in need, and I am excited to see the positive impact on
these patients.”
The Health Canada approval is based on the results of the
largest studies conducted to date in patients with obesity due to
BBS as well as POMC, PCSK1, and LEPR deficiencies. In Phase 3
trials in each of these diseases, setmelanotide achieved
statistically significant, clinically meaningful and sustained
reductions of body weight, represented by at least a 10% weight
loss from baseline at week 52. The trials met all primary endpoints
and key secondary endpoints among patients at 52 weeks on therapy.
Results from these trials were featured in several publications,
including the peer-reviewed journal The Lancet Diabetes and
Endocrinology. In clinical trials, IMCIVREE was generally
well-tolerated. Disturbance in sexual arousal, depression and
suicidal ideation, increased skin pigmentation and darkening of
pre-existing nevi, and benzyl alcohol toxicity in neonates and low
birth-weight infants may occur. The most common adverse reactions
were skin hyperpigmentation, injection site reactions and
nausea.
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,
the potential benefits of setmelanotide for patients with BBS or
POMC, PCSK1 and LEPR deficiencies, potential regulatory
submissions, approvals and timing thereof of setmelanotide, and our
business strategy and plans, including regarding commercialization
of IMCIVREE in Canada and other international regions. 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, failure to realize the anticipated
benefits of our acquisition of Xinvento B.V. or significant
integration difficulties related to the acquisition, and the other
important factors discussed under the caption “Risk Factors” in our
Annual Report on Form 10-K for the year ended December 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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