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FDA approves Imcivree in Bardet-Biedl syndrome.- Rhythm Pharma

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Published:18th Jun 2022

Rhythm Pharmaceuticals announced that the FDA has approved the Company’s supplemental New Drug Application (sNDA) for Imcivree (setmelanotide), a melanocortin-4 receptor (MC4R) agonist, for patients with Bardet-Biedl syndrome (BBS).


Imcivree was initially approved by the FDA in November 2020 for chronic weight management in adult and pediatric patients 6 years of age and older with obesity due to POMC, PCSK1 or LEPR deficiency. Imcivree’s label was updated today to include an FDA-approved test developed under a post-marketing commitment to confirm variants in POMC, PCSK1 or LEPR genes that are interpreted as pathogenic, likely pathogenic, or of uncertain significance (VUS).

Imcivree is not indicated for the treatment of patients with obesity due to suspected POMC, PCSK1 or LEPR deficiency with POMC, PCSK1 or LEPR variants classified as benign or likely benign, or other types of obesity not related to POMC, PCSK1 or LEPR deficiency, or BBS, including obesity associated with other genetic syndromes and general (polygenic) obesity.

The FDA application was based on data from Rhythm’s pivotal Phase III clinical trial, which was the largest and longest interventional clinical trial in BBS. In the clinical trial, Imcivree delivered early, significant and sustained weight reduction. The trial met its primary endpoint and all key secondary endpoints, with statistically significant reductions in weight and hunger at 52 weeks on therapy. 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.

Condition: Bardet-Biedl Syndrome
Type: drug

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