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
it has completed its supplemental New Drug Application (sNDA) to
the U.S. Food and Drug Administration (FDA) for IMCIVREE®
(setmelanotide) for the treatment of obesity and control of hunger
in adult and pediatric patients 6 years of age and older with
Bardet-Biedl syndrome (BBS) or Alström syndrome.
“This marks a major step in our efforts to bring IMCIVREE to
more patients and families living with rare genetic diseases of
obesity, including BBS, which affects approximately 1,500 to 2,500
people in the United States, and Alström syndrome, which affects
approximately 500 to 1,000 people worldwide,” said David Meeker,
M.D., Chairman, President and CEO of Rhythm. “Our application is
based on data from the pivotal Phase 3 trial in which treatment
with IMCIVREE produced clinically meaningful and statistically
significant reductions in body weight and in the unrelenting hunger
associated with these syndromes. The submission includes a series
of comprehensive individual patient narratives supporting our
belief that IMCIVREE has the potential to offer the first
therapeutic option for the early-onset, severe obesity and
unrelenting hunger that characterize these syndromes.”
The FDA typically has a 60-day filing review period to determine
whether the sNDA is sufficiently complete and acceptable for
filing. Rhythm has requested priority review for the application,
which, if granted, could provide a target FDA review period of
six-months from the date the sNDA is accepted. The Company also
expects to submit a Type II variation marketing authorization
application (MAA) to the European Medicines Agency (EMA) in the
fourth quarter of 2021, which also will cover both BBS and Alström
syndrome.
As first reported in December 2020, Rhythm’s Phase 3 trial of
setmelanotide in patients with BBS or Alström syndrome met its
primary endpoint and all key secondary endpoints, with
statistically significant and clinically meaningful reductions in
weight and hunger at 52 weeks on therapy. All primary endpoint
responders were patients with BBS; no patients with Alström
syndrome met the primary endpoint of more than 10 percent weight
loss.
Dr. Meeker continued, “While there are limited data for the
ultra-rare Alström syndrome population, there is clear evidence of
a marked and sustained weight loss in older children and adults,
and consistent reductions in BMI-Z score in younger patients. We
look forward to working closely with the FDA as we pursue this
additional approval.”
Rhythm also announced data in April 2021 at the Pediatric
Endocrine Society annual meeting from a predefined exploratory
endpoint showing the impact of setmelanotide on BMI-Z scores for
patients younger than 18 years old with BBS or Alström syndrome.
The BMI-Z score, or BMI standard deviation score, represents the
number of standard deviations from median BMI by child age and sex.
Setmelanotide was associated with statistically significant and
clinically meaningful reductions in BMI-Z scores. For 16 patients
younger than 18 years of age with BBS, the mean BMI-Z score was
reduced from 3.74 at baseline to 2.98 for a reduction of -0.76, or
-24.5 percent (p=0.0006).
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
cognitive impairment, polydactyly, renal dysfunction, hypogonadism,
and visual impairment. Clinical features of Alström syndrome may
include progressive visual and auditory impairment, insulin
resistance and Type 2 diabetes, hyperlipidemia, progressive kidney
dysfunction, cardiomyopathy, and short stature in adulthood.
Insatiable hunger, also known as hyperphagia, and severe obesity
beginning early in life may be common in people living with either
BBS or Alström syndrome. In the United States, the Company
estimates that BBS affects approximately 1,500 to 2,500 people and
that Alström syndrome affects approximately 500 people. Currently,
there are no approved therapies targeting the MC4 receptor pathway
for reducing body weight and hunger 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. The Company’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 by
the European Commission (EC) in July 2021 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
and EC-approved therapy for patients with these rare genetic
diseases of obesity. Rhythm is advancing a broad clinical
development program for setmelanotide in other rare genetic
diseases of obesity. The Company 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 deficiencies. The company is based 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, 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 Full Prescribing Information for IMCIVREE.
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. 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 June 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
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