2022-06-17| Trials & Approvals

Rhythm Pharmaceuticals Trumpets FDA Approval Of Obesity Drug For Bardet-Biedl Syndrome

by Joy Lin
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Rhythm Pharmaceuticals has announced the US FDA approval of Imcivree (setmelanotide) for Bardet-Biedl syndrome (BBS), a rare disease characterized by insatiable hunger (hyperphagia) and severe obesity that surfaces early in life. This marks another win for Rhythm’s MC4R approach for chronic weight management and obesity.

With the latest approval, Imcviree is indicated for adult and pediatric patients 6 years and older with obesity caused by rare diseases proopiomelanocortin (POMC), proprotein convertase subtilisin/ kexin type 1 (PCSK1) or leptin receptor (LEPR) deficiency, or BBS. 


Significant, Sustained Weight Reduction 


The FDA approval is based on data from Rhythm’s pivotal Phase 3 clinical trial assessing Imcivree in BBS. In the trial, Imcivree delivered early, significant and sustained weight reduction, meeting its primary endpoint and all key secondary endpoints.

52 weeks into the trial, the drug lowered BMI by an average of 7.9% without requirements for diet or exercise. When adjusted for placebo, the reduction in BMI was -4.5% in a 14-week double-blind placebo-controlled stage. At 52 weeks in patients 12 and over, there was a statistically significant mean reduction in hunger score (-2.1).

Imcivree works as a melanocortin-4 receptor (MC4R) agonist. The MC4R pathway regulates hunger, food intake, and energy usage. Impairments in the pathway due to genetic variations may cause excessive hunger and early-onset obesity. By restoring the function of the impaired pathway, Imcivree is intended to reduce hunger and weight in patients with rare genetic diseases of obesity. 

The drug is also approved in the EU and the UK for similar indications as the US. 


Side Effects of Imcivree


In a separate development, the FDA has issued a complete response letter for Imcivree for Alström syndrome, a rare cardiometabolic disease, in the US. 

The company will reevaluate the future of Imcivree for Alström syndrome, said Rhythm.

While generally well tolerated, Imcivree may cause nausea, disturb sexual arousal, increase depression and suicidal ideation, and increase skin pigmentation and darkening of pre-existing nevi. 


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