Rhythm Pharmaceuticals Announces Positive Opinion by the European Medicines Agency on Orphan Drug Designation for Setmelanoti...
July 29 2019 - 8:00AM
Rhythm Pharmaceuticals, Inc. (Nasdaq:RYTM), a biopharmaceutical
company focused on the development and commercialization of
therapeutics for the treatment of rare genetic disorders of
obesity, today announced that the European Medicines Agency’s
(EMA’s) Committee for Orphan Medicinal Products (COMP) has adopted
a positive opinion recommending setmelanotide for designation as an
orphan medicinal product for the treatment of patients with
Bardet-Biedl syndrome (BBS). People living with BBS may experience
an insatiable hunger, also known as hyperphagia, and severe obesity
beginning early in life. Rhythm is currently evaluating
setmelanotide in an ongoing pivotal Phase 3 trial in patients with
BBS and Alström syndrome.
“This positive opinion on orphan medicinal product designation
for setmelanotide in BBS, as well as our PRIority MEdicines (PRIME)
designation, reflects our commitment to working closely with the
EMA to advance setmelanotide for patients with BBS who have limited
treatment options for their hunger or obesity,” said Murray
Stewart, M.D., Chief Medical Officer of Rhythm. “We believe
setmelanotide has the potential to transform the treatment of BBS
and other rare genetic disorders of obesity by addressing
underlying defects in the melanocortin-4 receptor (MC4R) pathway.
This designation reflects the significant need for new therapies
for BBS and we look forward to working with the EMA to potentially
deliver setmelanotide to patients in Europe.”
The orphan medicinal product designation by the European
Commission is granted to medicines being developed for the
diagnosis, prevention or treatment of a life-threatening or
chronically debilitating condition that affects fewer than five in
10,000 people in the EU. This designation could allow for a number
of incentives, including protocol assistance, access to the
centralized authorization procedure, reduced regulatory fees, and a
ten-year period of market exclusivity in the EU after product
approval.
About Bardet-Biedl SyndromeBBS is an
ultra-rare, genetic disorder that affects multiple organ
systems. Clinical features may include and are not limited to
severe obesity, insatiable hunger, retinal degeneration,
polydactyly, kidney abnormalities, and developmental delays.
There is great variability in presentation and severity of these
features across individuals with BBS. Currently there are no
approved therapies for regulating hunger in BBS.
About SetmelanotideSetmelanotide is a potent
MC4R agonist in development for the treatment of rare genetic
disorders of obesity. Setmelanotide activates MC4R, part of the key
biological pathway that independently regulates energy expenditure
and appetite. Variants in genes within the MC4R pathway are
associated with unrelenting hunger and severe, early-onset obesity.
Rhythm is currently developing setmelanotide as a replacement
therapy for patients with monogenic defects upstream of MC4R, for
whom there are no effective or approved therapies. The FDA has
granted Breakthrough Therapy designation to setmelanotide for the
treatment of obesity associated with genetic defects upstream of
the MC4 receptor in the leptin-melanocortin pathway, which includes
POMC deficiency obesity, LEPR deficiency obesity, BBS and Alström
Syndrome. The European Medicines Agency has also granted PRIority
MEdicines (PRIME) designation for setmelanotide for the treatment
of obesity and the control of hunger associated with deficiency
disorders of the MC4R pathway.
About RhythmRhythm is a biopharmaceutical
company focused on the development and commercialization of
therapies for the treatment of rare genetic disorders of obesity.
Rhythm is currently evaluating the efficacy and safety of
setmelanotide, the company’s first-in-class MC4R agonist, in Phase
3 studies in patients with Pro-opiomelanocortin (POMC) deficiency
obesity, Leptin receptor (LEPR) deficiency obesity, Bardet-Biedl
syndrome, and Alström syndrome. The company is leveraging the
Rhythm Engine -- comprised of its Phase 2 basket study, TEMPO
Registry, GO-ID genotyping study and Uncovering Rare Obesity
program -- to improve the understanding, diagnosis and potentially
the treatment of rare genetic disorders of obesity. For healthcare
professionals, visit www.UNcommonObesity.com for more
information. For patients and caregivers,
visit www.LEADforRareObesity.com for more information.
The company is based in Boston, MA.
Forward-Looking Statements
This press release contains certain statements that are
forward-looking within the meaning of Section 27A of the
Securities Act of 1933, as amended, and Section 21E of the
Securities Exchange Act of 1934, as amended, and that involve risks
and uncertainties, including statements regarding Rhythm’s business
strategy and goals, possible benefits from receipt of orphan
medical product designation, and its expectations regarding
setmelanotide. Statements using words such as “expect”, “goal”,
“anticipate”, “believe”, “may”, “will”, “plan” 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, risks associated with data analysis and reporting, and
expenses, and other risks as may be detailed from time to time in
our Annual Reports on Form 10-K and quarterly reports on
Form 10-Q and other reports we file 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
Rhythm Pharmaceuticals (NASDAQ:RYTM)
Historical Stock Chart
From Jun 2024 to Jul 2024
Rhythm Pharmaceuticals (NASDAQ:RYTM)
Historical Stock Chart
From Jul 2023 to Jul 2024