Rhythm Completes Submission of SNDA for Imcivree in Bardet-Biedl and Alstrom Syndromes

By Michael Dabaie

Rhythm Pharmaceuticals Inc. said it completed its supplemental new drug application to the U.S. Food and Drug Administration for Imcivree for the treatment of obesity and control of hunger in adult and pediatric patients with Bardet-Biedl syndrome or Alstrom syndrome.

BBS and Alstrom syndrome are ultra-rare genetic diseases that affect multiple organ systems. Insatiable hunger and severe obesity beginning early in life may be common in people living with either BBS or Alstrom syndrome.

The FDA typically has a 60-day filing review period to determine whether the supplemental new drug application is sufficiently complete and acceptable for filing. Rhythm requested priority review for the application.

Rhythm said in December its Phase 3 trial of setmelanotide in patients with BBS or Alstrom syndrome met its primary objective and all key secondary endpoints, with statistically significant and clinically meaningful reductions in weight and hunger at 52 weeks on therapy. All primary endpoint responders were patients with BBS, and no patients with Alstrom syndrome met the primary endpoint of more than 10% weight loss, the company said.

"While there are limited data for the ultra-rare Alstrom syndrome population, there is clear evidence of a marked and sustained weight loss in older children and adults, and consistent reductions in BMI-Z score in younger patients," Rhythm Chairman and Chief Executive David Meeker said.

Write to Michael Dabaie at michael.dabaie@wsj.com

(END) Dow Jones Newswires

September 20, 2021 08:33 ET (12:33 GMT)

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