{"id":535843,"date":"2021-09-08T16:04:10","date_gmt":"2021-09-08T20:04:10","guid":{"rendered":"https:\/\/www.marketnewsdesk.com\/index.php\/rhythm-pharmaceuticals-announces-expanded-access-program-for-setmelanotide-for-patients-with-bardet-biedl-syndrome\/"},"modified":"2021-09-08T16:04:10","modified_gmt":"2021-09-08T20:04:10","slug":"rhythm-pharmaceuticals-announces-expanded-access-program-for-setmelanotide-for-patients-with-bardet-biedl-syndrome","status":"publish","type":"post","link":"https:\/\/www.marketnewsdesk.com\/index.php\/rhythm-pharmaceuticals-announces-expanded-access-program-for-setmelanotide-for-patients-with-bardet-biedl-syndrome\/","title":{"rendered":"Rhythm Pharmaceuticals Announces Expanded Access Program for Setmelanotide for Patients with Bardet-Biedl Syndrome"},"content":{"rendered":"

\nExpanded access program may allow access for eligible patients in the United States who have no authorized treatment options
\n<\/h2>\n
\n

BOSTON, Sept. 08, 2021 (GLOBE NEWSWIRE) — Rhythm Pharmaceuticals, Inc. (Nasdaq: RYTM), a commercial-stage biopharmaceutical company committed to transforming the care of people living with rare genetic diseases of obesity, today announced an Expanded Access Program (EAP) for setmelanotide for the treatment of eligible patients in the United States with severe obesity and hyperphagia due to Bardet-Biedl Syndrome (BBS). Rhythm expects to submit to the U.S. Food and Drug Administration (FDA) a supplemental new drug application (sNDA) for setmelanotide for the treatment of adult and pediatric patients 6 years of age and older with hunger and obesity due to BBS by the end of September.<\/p>\n

\u201cBBS is a serious disease with significant unmet needs,\u201d said David Meeker, M.D., Chair, President and Chief Executive Officer of Rhythm. \u201cFollowing the completion of our Phase 3 clinical trial and as we advance through the regulatory review process, we are pleased to make setmelanotide available through an expanded access program to eligible patients in the United States who are living with BBS. This program reflects our commitment to delivering therapies for rare genetic diseases of obesity as rapidly as possible.\u201d<\/p>\n

The FDA\u2019s expanded access regulations are designed to facilitate access to an investigational therapy to treat patients who are unable to participate in clinical trials and have serious or immediately life-threatening diseases or conditions for which there are no comparable or satisfactory alternative treatment options.<\/p>\n

If a treating physician believes setmelanotide may be the only therapeutic option for a patient with severe obesity and hyperphagia due to BBS, the physician can request additional information by contacting ExpandedAccess@rhythmtx.com<\/a>.<\/p>\n

\n About\u00a0Rhythm Pharmaceuticals<\/strong>
\n
Rhythm is a commercial-stage biopharmaceutical company committed to transforming the treatment paradigm for people living with rare genetic diseases of obesity. The Company\u2019s precision medicine, IMCIVREE\u00ae<\/strong><\/sup>\u00a0(setmelanotide), was approved in\u00a0November 2020\u00a0by the\u00a0U.S. Food and Drug Administration\u00a0(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\u00a0European Commission\u00a0(EC) in\u00a0July 2021\u00a0for 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\u2014now with approximately 37,500 sequencing samples\u2014to improve the understanding, diagnosis and care of people living with severe obesity due to certain genetic deficiencies. The company is based in\u00a0Boston, MA.<\/p>\n

\n IMCIVREE<\/strong>
\n
\n \u00ae<\/strong>
\n <\/sup>
\n \u00a0(setmelanotide) Indication<\/strong>
\n
In\u00a0the 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\u00a0POMC<\/em>,\u00a0PCSK1<\/em>, or\u00a0LEPR<\/em>\u00a0genes that are interpreted as pathogenic, likely pathogenic, or of uncertain significance (VUS).<\/p>\n

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.<\/p>\n

\n Limitations of Use<\/strong>
\n
IMCIVREE is not indicated for the treatment of patients with the following conditions as IMCIVREE would not be expected to be effective:<\/p>\n