FDA Delays PDUFA Date of Setmelanotide for Rare Obesity Syndromes

The US Food and Drug Administration (FDA) issued a 3-month extension of review period for setmelanotide (IMCIVREE), which originally had a Prescription Drug User Fee Act (PDUFA) date set for today, March 16. The updated date has been revised to June 16, 2022.

The setmelanotide supplemental New Drug Application (sNDA) was designated for the treatment of obesity and hunger control in adult and pediatric patients (6 years and older) with Bardet-Biedl syndrome (BBS) or Alström syndrome.

In 2020, setmelanotide was the first-ever therapy for chronic weight management for adult and pediatric patients with obesity caused by proopiomelanocortin (POMC), proprotein convertase subtilisin/kexin type 1 (PCSK1) or leptin receptor (LEPR) deficiency.

According to the FDA, more time was needed to review the sNDA. Last month, additional subgroup analyses regarding the clinical efficacy data from the phase 3 pivotal trial in BBS and Alström syndrome was requested from Rhythm Pharmaceuticals.

Rhythm announced positive interim data from the long-term extension study in February along with plans to present the detailed results later this year. In the extension, setmelanotide was generally well-tolerated and no new safety signals were observed.

Of the 19 patients who received treatment for 24 months, the mean reduction in body mass index (BMI) from baseline was -14.3%. The mean percent of body weight reduction in patients 18 or older was -14.9%. Among patients younger than 18 years of age, the mean reduction in BMI Z score was -0.72.

“There is a significant need for an effective therapy for patients with BBS for the hyperphagia, or pathological hunger, and severe obesity that manifests in childhood,” Bob Haws, MD, Director of Clinical Research Center, Marshfield Clinic Research Institute, Director of the Center of Excellence, Bardet-Biedl Syndrome, said in a statement. “With these long-term data, we are encouraged to see that the clinically meaningful weight loss and reduction of hyperphagia in patients at one year on therapy was sustained and even deepened at two years on setmelanotide, without specific dietary and exercise support.”