Rhythm Pharmaceuticals, Inc. (Nasdaq: RYTM), a 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 Great Britain's Medicines & Healthcare products Regulatory Agency (MHRA) has expanded the marketing authorization for IMCIVREE (setmelanotide) to include the treatment of obesity and control of hunger associated with genetically confirmed Bardet-Biedl syndrome (BBS) in adult and pediatric patients 6 years of age and older.
Authorization in Great Britain for BBS is a significant milestone on the path toward delivering IMCIVREE globally to treat rare MC4R pathway-related obesities,' said Yann Mazabraud, Executive Vice President, Head of International at Rhythm Pharmaceuticals. 'Based on the early reception to our BBS launch in the United States and European Union, it's clear that BBS patients and their families have been waiting for a precision medicine that can effectively address both hyperphagia and severe obesity. We look forward to building on our ongoing momentum as we work with health care authorities and prepare to deliver our precision therapy to patients living in Great Britain and advance our country-by-country strategy to achieve broader market access for IMCIVREE.'
IMCIVREE was selected for evaluation as a 'Highly Specialised Technology' by the National Institute for Health and Care Excellence (NICE). The Company plans to work closely with the United Kingdom's National Health Service (NHS) to finalize guidance for coverage of IMCIVREE.
BBS is a rare genetic disease that affects approximately 2,500 people in the European Union (EU) and United Kingdom (UK). People living with BBS may experience insatiable hunger, also known as hyperphagia, and severe obesity beginning early in life. Approximately 1,500 patients have been diagnosed with BBS and are now being cared for at academic settings in the EU and UK.
Health Canada Accepts for Review New Drug Submission for Setmelanotide
Rhythm also announced today that Health Canada has accepted for review its New Drug Submission (NDS) for setmelanotide, indicated in adults and pediatric patients 6 years of age and older with impairments in the MC4R pathway due to genetic diseases, for the treatment of obesity and control of hunger in BBS or biallelic pro-opiomelanocortin (POMC), proprotein convertase subtilisin/kexin type 1 (PCSK1), or leptin receptor (LEPR) deficiency. Health Canada granted Priority Review of the NDS, which shortens the submission review performance target to 180 days, in comparison to 300 days for non-priority review.
We are pleased that Health Canada has accepted our NDS and look forward to working closely with the agency throughout the review process,' said Carol Stiff, Rhythm's General Manager, Canada. 'We are now establishing business operations in Canada, leveraging our international resources and the robust foundation we have built in the United States and Europe to inform our future potential launch.'
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) pathway diseases. Rhythm's precision medicine, setmelanotide, is approved 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 monogenic or syndromic obesity due to pro-opiomelanocortin (POMC), proprotein convertase subtilisin/kexin type 1 (PCSK1) or leptin receptor (LEPR) deficiency confirmed by genetic testing, or patients with a clinical diagnosis of Bardet-Biedl syndrome (BBS). The European Commission (EC) and Great Britain's Medicines & Healthcare Products Regulatory Agency (MHRA) have authorized setmelanotide for the treatment of obesity and the control of hunger associated with genetically confirmed BBS or genetically confirmed loss-of-function biallelic POMC, including PCSK1, deficiency or biallelic LEPR deficiency in adults and children 6 years of age and above. 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 45,000 sequencing samples-to improve the understanding, diagnosis and care of people living with severe obesity due to certain genetic deficiencies. Rhythm's headquarters is in Boston, MA.
2022 Rhythm Pharmaceuticals, Inc. All Rights Reserved. Rhythm, IMCIVREE, [etc.] and their corresponding logos are trademarks of Rhythm Pharmaceuticals, Inc.
Setmelanotide Indication
In the United States, setmelanotide is indicated for chronic weight management in adult and pediatric patients 6 years of age and older with monogenic or syndromic obesity due to POMC, PCSK1 or LEPR deficiency as determined by an FDA-approved test demonstrating variants in POMC, PCSK1 or LEPR genes that are interpreted as pathogenic, likely pathogenic, or of uncertain significance (VUS) or BBS.
In the European Union and Great Britain, setmelanotide is indicated for the treatment of obesity and the control of hunger associated with genetically confirmed Bardet-Biedl syndrome (BBS) or genetically confirmed loss-of-function biallelic pro-opiomelanocortin (POMC), including PCSK1, deficiency or biallelic leptin receptor (LEPR) deficiency in adults and children 6 years of age and above.
Limitations of Use
In the United States and Europe, setmelanotide should be prescribed and supervised by a physician with expertise in obesity with underlying genetic etiology.
Setmelanotide is not indicated for the treatment of patients with the following conditions as setmelanotide 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, or BBS, including obesity associated with other genetic syndromes and general (polygenic) obesity.
Forward-Looking Statements
This press release contains forward-looking statements within the meaning of the U.S. 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 setmelanotide for patients with BBS or monogenic or syndromic obesity due to POMC, PCSK1 or LEPR deficiency confirmed by genetic testing, and our expectations surrounding potential regulatory submissions, approvals and timing thereof, and our business strategy and plans, including regarding commercialization of setmelanotide in Great Britain, Canada, Europe, the United States 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, 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, 2022 and our other filings with the U.S. 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.
Contact:
David Connolly
Tel: 857-264-4280
Email: dconnolly@rhythmtx.com
RHYTHM PHARMACEUTICALS, INC. 
