Rhythm Pharmaceuticals Receives European Medicines Agency PRIME Designation for Setmelanotide in Rare Genetic Disorders of Obesity

Rhythm Pharmaceuticals (NASDAQ:RYTM) has announced that the European Agency (EMA) has granted PRIority MEdicines (PRIME) designation for the company’s melanocortin-4 receptor (MC4R) for the treatment of obesity and the control of hunger associated with deficiency disorders of the MC4R pathway.

As quoted in the press release:

The PRIME program was launched by the EMA in 2016 to provide early and enhanced support to optimize the development of eligible medicines, speed up their evaluation, and contribute to timely patient access. To be eligible for PRIME, medicines must address an unmet medical need and preliminary data must be available showing the potential to address this need and bring a major therapeutic advantage to patients.

“We are pleased to receive PRIME designation, which allows us the opportunity to engage more closely with the EMA and potentially accelerate our clinical development of setmelanotide in the European Union,” said Keith Gottesdiener, M.D., Chief Executive Officer of Rhythm Pharmaceuticals. “The receipt of PRIME designation for setmelanotide further validates the fact that people living with rare genetic disorders of obesity are severely underserved by current treatments, which fail to address underlying disease biology. We look forward to working closely with the EMA through our pivotal-stage clinical research to deliver a potentially transformative treatment option to patients.”

Rhythm has completed pivotal enrollment in two ongoing Phase 3 clinical trials evaluating setmelanotide in pro-opiomelanocortin (POMC) and leptin receptor (LEPR) deficiency obesity and expects to report initial data in the third quarter of 2019. The Company is also evaluating setmelanotide in four additional rare genetic disorders of obesity, including Bardet-Biedl Syndrome (BBS), Alström Syndrome, POMC and other MC4R pathway heterozygous deficiency obesities, and POMC epigenetic disorders. Rhythm plans to initiate a Phase 3 trial of setmelanotide in BBS and Alström Syndrome by the end of 2018, and intends to continue enrolling patients with POMC and other MC4R pathway heterozygous deficiency obesities, as well as POMC epigenetic disorders, in its ongoing Phase 2 basket studies, in order to identify those patients most likely to benefit from setmelanotide treatment.

Click here to read the full press release.