FDA Approved Weight-Management Treatment for Bardet–Biedl Syndrome
Late last week, the FDA approved a weight-management treatment for patients with Bardet–Biedl Syndrome.
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- The FDA recently approved the Imcivree (setmelanotide) injection as a weight-management treatment for adults and children over 6 with Bardet–Biedl Syndrome (BBS). BBS is a relatively uncommon genetic disorder with a broad range of symptoms.
The most common symptoms are excessive weight gain and obesity, which often develop in infancy.
According to the European Journal of Human Genetics, one-third of patients with BBS develop obesity by their first birthday. Later in life, they are also more likely to develop type 2 diabetes. It is thought that there is a potential link between the two.
In the press release, the FDA notes that Imcivree was first approved in November of 2020 for weight management in patients older than 6 with proopiomelanocortin (POMC), proprotein convertase subtilisin/kexin type 1 (PCSK1), or leptin receptor (LEPR) deficiency.
In the announcement by Rhythm Pharmaceuticals — the company that manufactures Imcivree — the drug is identified as a melanocortin-4 receptor (MC4R) agonist. MC4R plays an integral role in weight management. These receptors are a part of hunger, satiety, and weight-control neuronal pathways.
Patients with BBS have mutations in the gene that codes for the MC4R receptors. By acting as an agonist for said receptors, Imcivree activates them more readily, thus helping control appetite.
The study looked at patients over 6 with BBS and a BMI over 30. Patients were given Imcivree for one year and evaluated on BMI and hunger levels.
At the conclusion of the study, patients on Imcivree saw an average 7.9% decrease in their BMI. Furthermore, 39% of patients saw a reduction of 10% or more in their BMI. Simultaneously, patients on the drug reported an overall decrease in their hunger scores.
This FDA approval could be a monumental change in the management of BBS symptoms as it is the only drug currently approved for weight management in this condition. There are implications that the use of this medication could also decrease rates of type 2 diabetes in BBS patients.
The FDA and Rhythm Pharmaceuticals put forth safety information associated with using Imcivree, as it may have adverse side effects, including sexual arousal disturbances, depression, and suicidal thoughts.
Despite these potential side effects, many members of the BBS community are hopeful regarding its impact on BBS Patients.
“Challenges in managing weight and hunger have dramatically negative effects on health, well-being, and quality of life for those living with BBS, and we are thrilled finally to have a new treatment option to address this significant unmet need,” remarked Timothy Ogden, President, BBS Foundation and Family Association in the Rhythm Pharmaceuticals report.
