EMA Favors Naming Setmelanotide an Orphan Medicine for Bardet-Biedl Syndrome
A branch of the European Medicines Agency (EMA) has recommended that Rhythm‘s setmelanotide be designated an orphan medicine to treat people with Bardet-Biedl syndrome (BBS), a rare genetic disorder of obesity.
The positive opinion of EMA’s Committee for Orphan Medicinal Products (COMP) puts setmelanotide closer to a range of incentives that can potentially accelerate its development and approval.
Rhythm is running several clinical trials to test setmelanotide’s safety and efficacy in a number of genetic obesity disorders. One is a pivotal Phase 3 trial (NCT03746522) evaluating setmelanotide in people with BBS and Alström syndrome. The trial has sites open in the U.S. and Canada.
“This positive opinion on orphan medicinal product designation for setmelanotide in BBS, as well as our PRIority MEdicines (PRIME) designation, reflects our commitment to working closely with the EMA to advance setmelanotide for patients with BBS who have limited treatment options for their hunger or obesity,” Murray Stewart, MD, Rhythm’s chief medical officer, said in a press release.
“We believe setmelanotide has the potential to transform the treatment of BBS and other rare genetic disorders of obesity by addressing underlying defects in the melanocortin-4 receptor (MC4R) pathway. This designation reflects the significant need for new therapies for BBS and we look forward to working with the EMA to potentially deliver setmelanotide to patients in Europe,” he added.
The European Commission (EU) grants an orphan designation to medicinal products that address an unmet need or provide a significant benefit compared to other available products for life-threatening or chronically debilitating conditions that are rare.
The designation opens the door to a number of incentives to facilitate development of new medicines, including protocol assistance, reduced regulatory fees, access to the centralized authorization procedure, and a 10-year period of market exclusivity in the EU after approval.
Rhythm is developing setmelanotide (RM-493) — an activator of MC4R signals — for patients with genetic defects that hamper MC4R pathway that works to control sensations of hunger.
The FDA designated setmelanotide a breakthrough therapy for the treatment of several genetic obesity disorders, including BBS, in 2017. Last year, EMA also granted setmelanotide PRIority MEdicines (PRIME) designation for the treatment of obesity associated with deficiency disorders of the MC4R pathway.
Other ongoing studies testing setmelanotide in people with genetic obesity include: a Phase 2/3 trial (NCT03013543) evaluating the compound for people with POMC deficiency, LEPR deficiency, BBS, Alström syndrome, and Smith-Magenis syndrome; a Phase 3 trial for LEPR deficiency (NCT03287960); and a Phase 2/3 trial for POMC deficiency (NCT02896192). All these trials are currently enrolling patients.