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The FDA also issued a complete response letter for Imcivree for use in patients with Alstrom syndrome, another genetic rare disease that can cause obesity.
The FDA has approved Rhythm Pharmaceuticals’ supplemental new drug application (sNDA) for Imcivree (setmelanotide) for patients with Bardet-Biedl syndrome (BBS). It is indicated for chronic weight management in adults and children 6 years old and older with obesity in patients with BBS.
“It is important to understand that the early-onset, severe obesity and pathologic hunger experienced by many people living with BBS can be debilitating, especially when you consider the impact of food-seeking behavior on families,” Timothy Ogden, president, BBS Foundation and Family Association, said in a press release.
Bardet Biedl Syndrome is a rare genetic disorder that impacts about 1,500 to 2,500 people in the United States. It can have variable symptoms that include retinal degeneration, obesity, reduced kidney function, or extra fingers or toes. Weight is generally normal at birth but infants with BBS quickly gain weight, with fat distributed in the abdomen and chest. About 90% of patients develop obesity. This can lead to diabetes and complications related to the heart and blood vessels.
Imcivree is a melanocortin-4 receptor (MC4R) agonist. MC4R is part of the key biological pathway that regulates hunger, caloric intake and energy expenditure. Variants in genes may impair the function of the MC4R pathway.
The approval was based on data from a phase 3 trial of 31 patients where Imcivree met primary and secondary endpoints. In patients six years and older, the mean change in BMI was -7.9% without diet or exercise, and there was a statistically significant change in hunger score.
At the same time, the FDA issued a complete response letter for Imcivree for use in patients with Alstrom syndrome, another genetic rare disease that can cause obesity from a mutation in the ALMS gene
The FDA initially approved Imcivree in November 2020 for adult and pediatric patients 6 years old and older with monogenic or syndromic obesity due to proopiomelanocortin (POMC), proprotein convertase subtilisin/kexin type 1 (PCSK1) or leptin receptor (LEPR) deficiency. Deficiency in POMC, PCSK1, and LEPR also affect the MC4R pathway.