The US Food and Drug Administration (FDA) has accepted the supplemental new drug application (sNDA) of Rhythm Pharmaceuticals for Imcivree (setmelanotide) to treat certain obesity disorders.
Imcivree, a melanocortin-4 receptor (MC4R) agonist, is being evaluated for treating obesity linked to Bardet-Biedl syndrome (BBS) or pro-opiomelanocortin (POMC) deficiencies.
This includes proprotein convertase subtilisin/kexin type 1 (PCSK1) and leptin receptor (LEPR) deficiencies, in children as young as two years old.
The FDA has granted priority review to the sNDA and set a Prescription Drug User Fee Act (PDUFA) goal date of 26 December 2024.
Previously, the biopharmaceutical company received a positive recommendation from the National Institute for Health and Care Excellence (NICE) for Imcivree label expansion.
The sNDA submission is supported by data from a one-year, multi-centre, open-label Phase 3 trial involving 12 patients aged two to under six years old.
These patients had obesity resulting from biallelic POMC/PCSK1 or LEPR deficiencies, or a clinical diagnosis of BBS.
The late-stage trial addressed hyperphagia and severe obesity associated with rare genetically-caused MC4R pathway disorders, which can manifest early in life.
Results indicated that treatment with setmelanotide met the primary endpoint, demonstrating a mean reduction of 3.04 in BMI-Z score, a measure of deviations from normal body mass index.
The findings also showed an 18.4% mean reduction in body mass index (BMI).
Rhythm Pharmaceuticals CEO and president David Meeker said: “Imcivree has been approved for more than two years and prescribed to hundreds of patients six years old and older, and we believe that treating patients at an even earlier age will positively affect the lives of these children and their families.”
Last month, the European Commission (EC) expanded the marketing authorisation for Imcivree to include children as young as two years old with obesity due to BBS or deficiencies in POMC/PCSK1 or LEPR.
In the US, the MC4R agonist is currently approved for chronic weight management in both adult and paediatric patients aged six years and older.
Additionally, Boston-based Rhythm Pharmaceuticals is advancing a clinical development programme for setmelanotide targeting other rare diseases.
The pharma company is also exploring investigational MC4R agonists, LB54640 and RM-718, and developing a preclinical suite of small molecules aimed at treating congenital hyperinsulinism.
Rhythm Pharmaceuticals acquired the global rights to LB-54640 from LG Chem Life Sciences in January 2024.
