FDA Approves Weight Management Drug Tested by Marshfield Clinic Health System

Monday, June 27th, 2022 -- 10:01 AM

The U.S. Food and Drug Administration has approved a chronic weight management drug for patients with the rare disease Bardet-Biedl syndrome.

Marshfield Clinic Health System is the only health system in the nation that provides comprehensive care for patients with BBS. BBS is caused by a mutation in a number of different genes that affects the way the body’s cells communicate with each other, ultimately affecting tissue growth during fetus development.

The result includes organs that form in the wrong position internally, extra fingers and toes, impaired vision, kidney problems, obesity and developmental delays.

Marshfield Clinic has participated in several clinical trials with Rhythm Pharmaceuticals, a biopharmaceutical company aimed at developing and commercializing therapies for the treatment of rare genetic diseases of obesity.

The FDA approved June 16 a supplemental indication for Imcivree (setmelanotide) injection for chronic weight management in adult and pediatric patients 6 years of age and older with obesity due to BBS.

Setmelanotide is the first drug approved specifically for chronic weight management in patients with BBS, a rare genetic disease associated with early-onset childhood obesity.

In clinical trials, IMCIVREE was generally well-tolerated. Disturbance in sexual arousal, depression and suicidal ideation, increased skin pigmentation and darkening of pre-existing nevi, and benzyl alcohol toxicity in neonates and low birth-weight infants may occur.

The most common adverse reactions were skin hyperpigmentation, injection site reactions and nausea. In March 2017, Marshfield Clinic gave the first dose of setmelanotide for chronic weight management.

Our body produces the hormone leptin that is important in telling our brain that we are full and do not need to eat. In those with BBS, leptin is unable to communicate with the part of the brain that controls appetite, which leads to hyperphagia, the unsatisfied drive to consume food.

What this drug does is bypass that signal disruption. It then goes to the second messenger and transmits the signal to the brain to stop eating. Marshfield Clinic Research Institute continued to lead the way by enrolling the most participants in the Rhythm-sponsored study and setting other landmarks such as enrolling the youngest participant.

BBS affects fewer than 3,000 individuals in the U.S. Because it is so rare, BBS may not be recognized early. This may delay the start of treatment and support for at-risk families.

BBS is inherited by both parents passing on one BBS gene to their child and evolves slowly throughout the first decade of life, although there is a lot of variability.

Under the direction of recently retired Robert Haws, M.D., the Center of Excellence for Bardet-Biedl Syndrome at Marshfield Clinic has become a renowned resource for BBS care and treatment.

Patients from across the world come to Marshfield Clinic to see nephrologists, dieticians, dermatologists, ophthalmologists, cardiologists, psychologists and other specialties with extensive familiarity of BBS for comprehensive care and treatment for this disease.

In addition, Marshfield Clinic Research Institute also is the home of the Clinical Registry Investigating Bardet-Biedl Syndrome (CRIBBS), the largest worldwide registry examining the long-term health of individuals with BBS.

The mission of CRIBBS is to promote research that will improve the health and quality of life of individuals affected by BBS. The research team collects health information of individuals with BBS so it can be used in many scientific endeavors.

Feel free to contact us with questions and/or comments.