Rhythm Pharmaceuticals has secured approval from the US Food and Drug Administration (FDA) for its supplemental New Drug Application (sNDA) for IMCIVREE (setmelanotide), a melanocortin-4 receptor (MC4R) agonist, for patients with Bardet-Biedl syndrome (BBS).
With this FDA approval, IMCIVREE is indicated for chronic weight management in adult and pediatric patients aged six years and older with monogenic or syndromic obesity due to proopiomelanocortin (POMC), proprotein convertase subtilisin/kexin type 1 (PCSK1) or leptin receptor (LEPR) deficiency, or BBS.
Rhythm chair, president and CEO David Meeker said: “This FDA approval represents a significant milestone for Rhythm, validating our strategy of developing IMCIVREE for people with hyperphagia and severe obesity caused by rare MC4R-pathway diseases and allowing us to provide our precision therapy to an established community of patients living with BBS and their families who are eagerly awaiting a new treatment option.
“Leveraging the robust infrastructure we put in place following the initial approval of IMCIVREE for obesity due to biallelic POMC, PCSK1 or LEPR deficiency and our new high-touch patient support services to assist patients throughout the journey from diagnosis to ongoing treatment, we are able to make IMCIVREE available for BBS immediately. We look forward to delivering this important medicine to the growing community of patients and families in need of options that can effectively address the obesity and hyperphagia that affect many people living with BBS.”
FDA had initially approved IMCIVREE in November 2020 for chronic weight management in adult and pediatric patients aged six years and older with obesity due to POMC, PCSK1 or LEPR deficiency.
With the latest development, IMCIVREE’s label has been updated 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 to treat patients with obesity because of suspected POMC, PCSK1 or LEPR deficiency with POMC, PCSK1 or LEPR variants categorised as benign or likely benign, or other kinds of obesity not associated to POMC, PCSK1 or LEPR deficiency, or BBS, including obesity related with other genetic syndromes and general (polygenic) obesity.
During clinical studies, IMCIVREE was found to be generally well-tolerated.
The most common adverse reactions reported were skin hyperpigmentation, injection site reactions and nausea. There may be 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.
A rare genetic disease, BBC impacts around 1,500-2,500 people in the US.
People suffering with BBS may feel insatiable hunger, also known as hyperphagia, and severe obesity starting early in life.
BBS may also be linked with cognitive impairment, polydactyly, renal dysfunction, hypogonadism, and visual impairment.
In the clinical trial, IMCIVREE delivered significant, early and sustained reduction in weight.
Furthermore, the trial achieved its primary endpoint and all key secondary endpoints, with significant reductions seen statistically in weight and hunger at 52 weeks on therapy.
BBS Foundation and Family Association president Timothy Ogden said: “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.
“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.”