EU Expands Marketing Authorization for Rhythm’s Imcivree to Treat Obesity and Control of Hunger in BBS

The European Commission has expanded the marketing authorization for Rhythm Pharmaceuticals’ Imcivree (setmelanotide) to include the treatment of obesity and control of hunger associated with genetically confirmed Bardet-Biedl syndrome in adult and pediatric patients 6 years of age and older.

Photo: David Meeker, chairman, president, and CEO of Rhythm

Bardet-Biedl syndrome (BBS) is a rare genetic disease that affects approximately 2,500 people in the European Union and United Kingdom. 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 European Union and United Kingdom.

“Patients and families have long endured a significant need for a therapy to address the burdens of hyperphagia and severe obesity often associated with BBS,” said Philip Beales, University College London, UCL Great Ormond Street Institute of Child Health. “Having new treatment options available can help address the physical and emotional aspects of this devastating disease, offering patients and their families relief and improved quality of life.”

Under the terms of the Revenue Interest Financing Agreement with HealthCare Royalty Partners announced on June 16, 2022, Rhythm is now eligible to receive an additional investment of $37.5 million following EC marketing authorization for Imcivree (setmelanotide) for BBS. Rhythm had received an initial investment amount of $37.5 million from HealthCare Royalty as a result of the approval of setmelanotide by the U.S. Food and Drug Administration, and the company remains eligible for an additional investment amount of $25 million, which would be payable upon the achievement of certain agreed sales milestones in 2023.

In the European Union, 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.

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.

Rhythm also said that Health Canada has granted Priority Review for Rhythm’s New Drug Submission for setmelanotide, indicated in adult 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.

Priority Review shortens Health Canada’s submission review performance target to 180 days, in comparison to 300 days for non-priority review. Priority Review may be granted for drug submissions in Canada for a serious, life-threatening or severely debilitating disease or condition for which there is substantial evidence of clinical effectiveness that the drug provides effective treatment, prevention or diagnosis of a disease or condition for which no drug is presently marketed in Canada or a significant increase in efficacy and/or significant decrease in risk such that the overall benefit/risk profile is improved over existing therapies, preventatives or diagnostic agents for a disease or condition that is not adequately managed by a drug marketed in Canada.

“These two important regulatory milestones underscore the global unmet need to treat the hyperphagia and severe obesity associated with these rare MC4R pathway diseases,” said David Meeker, chairman, president, and CEO of Rhythm. “We are working closely with Health Technology Assessment bodies and payers throughout the EU on a country-by-country basis to achieve market access and reimbursement in order to make IMCIVREE available to eligible patients as rapidly as possible. Taken together with the recent FDA approval for BBS in June, we are making significant advances in our mission of delivering the first precision medicine for MC4R pathway diseases to patients across the world.”

Author: Rare Daily Staff