Rhythm Pharmaceuticals, Inc. (Nasdaq: RYTM), a
biopharmaceutical company aimed at developing and commercializing
therapies for the treatment of rare genetic diseases of obesity,
today announced that the U.S. Food and Drug Administration (FDA)
has accepted for filing the company’s supplemental New Drug
Application (sNDA) for IMCIVREE® (setmelanotide), a melanocortin-4
receptor (MC4R) agonist, for patients with Bardet-Biedl syndrome
(BBS) or Alström syndrome. The FDA granted Priority Review of the
sNDA and assigned a Prescription Drug User Fee Act (PDUFA) goal
date of March 16, 2022.
“The acceptance for filing of our supplemental application by
the FDA marks an important step in our efforts to address the unmet
needs and bring IMCIVREE to patients and families living with
Bardet-Biedl syndrome and Alström syndrome,” said Linda Shapiro,
M.D., Ph.D., Chief Medical Officer of Rhythm Pharmaceuticals. “The
FDA’s decision to grant Priority Review to the application aligns
with our belief in the potential of IMCIVREE to deliver clinically
meaningful and statistically significant reductions in body weight
and hunger for patients with BBS and Alström syndrome while also
substantially improving quality of life for these patients and
their families.”
A Priority Review designation is granted by the FDA for the
evaluation of drug applications that may offer significant
improvements in the safety or effectiveness of the treatment,
prevention or diagnosis of a serious disease. Under the FDA’s
current PDUFA review goals, for an application granted Priority
Review, the FDA aims to take action on such application within six
months of receipt, compared to 10 months under standard review.
About Bardet-Biedl and Alström SyndromesBBS and
Alström syndrome are ultra-rare genetic diseases that affect
multiple organ systems. Clinical features of BBS may include
early-onset severe obesity, hyperphagia, cognitive impairment,
polydactyly, renal dysfunction, hypogonadism, and/or visual
impairment. Clinical features of Alström syndrome may include
early-onset severe obesity, hyperphagia, progressive visual and
auditory impairment, insulin resistance and Type 2 diabetes,
hyperlipidemia, progressive kidney dysfunction, cardiomyopathy, and
short stature in adulthood. Hyperphagia is an insatiable hunger
that is a common feature in both of these diseases and is
potentially related to impaired signaling via the MC4R pathway in
the hypothalamus.
Rhythm estimates that BBS affects approximately 1,500 to 2,500
people and that Alström syndrome affects approximately 500 people
in the United States, with a similar prevalence estimate
in Europe. Currently, there are no approved therapies
targeting the MC4R pathway to address the early-onset severe
obesity and hyperphagia in BBS or Alström syndrome.
About Rhythm PharmaceuticalsRhythm is a
commercial-stage biopharmaceutical company committed to
transforming the treatment paradigm for people living with rare
genetic diseases of obesity. Rhythm’s precision medicine, IMCIVREE
(setmelanotide), was approved in November 2020 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 obesity due to POMC, PCSK1 or LEPR deficiency
confirmed by genetic testing and in July and September 2021,
respectively, by the European Commission (EC) and Great
Britain’s Medicines & Healthcare Products Regulatory
Agency (MHRA) 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 with these rare genetic diseases of obesity.
The Company submitted a supplemental New Drug Application (sNDA) to
the FDA in September 2021 and submitted a Type II
variation application to the European Medicines
Agency in October 2021 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 or Alström syndrome in both the United
States and 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 37,500 sequencing samples --
to improve the understanding, diagnosis and care of people living
with severe obesity due to certain genetic variants. Rhythm’s
headquarters is in Boston, MA.
IMCIVREE®
(setmelanotide) IndicationIn the United States,
IMCIVREE is indicated for chronic weight management in adult and
pediatric patients 6 years of age and older with obesity due to
proopiomelanocortin (POMC), proprotein convertase subtilisin/kexin
type 1 (PCSK1), or leptin receptor (LEPR) deficiency. The condition
must be confirmed by genetic testing demonstrating variants
in POMC, PCSK1, or LEPR genes that are
interpreted as pathogenic, likely pathogenic, or of uncertain
significance (VUS).
In 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.
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, including obesity associated with other genetic
syndromes and general (polygenic) obesity.
Important Safety Information
WARNINGS AND PRECAUTIONS
Disturbance in Sexual Arousal: Sexual
adverse reactions may occur in patients treated with IMCIVREE.
Spontaneous penile erections in males and sexual adverse reactions
in females occurred in clinical studies with IMCIVREE. Instruct
patients who have an erection lasting longer than 4 hours to seek
emergency medical attention.
Depression and Suicidal Ideation: Some
drugs that target the central nervous system, such as IMCIVREE, may
cause depression or suicidal ideation. Monitor patients for new
onset or worsening of depression. Consider discontinuing IMCIVREE
if patients experience suicidal thoughts or behaviors.
Skin Pigmentation and Darkening of Pre-Existing
Nevi: IMCIVREE may cause generalized increased skin
pigmentation and darkening of pre-existing nevi due to its
pharmacologic effect. This effect is reversible upon
discontinuation of the drug. Perform a full body skin examination
prior to initiation and periodically during treatment with IMCIVREE
to monitor pre-existing and new skin 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.
ADVERSE REACTIONS
- The most common adverse reactions (incidence ≥23%) were
injection site reactions, skin hyperpigmentation, nausea, headache,
diarrhea, abdominal pain, back pain, fatigue, vomiting, depression,
upper respiratory tract infection, and spontaneous penile
erection.
USE IN SPECIFIC POPULATIONSDiscontinue IMCIVREE
when pregnancy is recognized unless the benefits of therapy
outweigh the potential risks to the fetus.
Treatment with IMCIVREE is not recommended for use while
breastfeeding.
To report SUSPECTED ADVERSE REACTIONS, contact Rhythm
Pharmaceuticals at +1 (833) 789-6337 or FDA at 1-800-FDA-1088
or www.fda.gov/medwatch.
See U.S. Full Prescribing Information, EU
SmPC and MHRA SmPC for IMCIVREE.
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. 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, 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
September 30, 2021 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
Rhythm Pharmaceuticals (NASDAQ:RYTM)
Historical Stock Chart
From Aug 2024 to Sep 2024
Rhythm Pharmaceuticals (NASDAQ:RYTM)
Historical Stock Chart
From Sep 2023 to Sep 2024