Rhythm announces pre-marketing early access authorization for setmelanotide

Rhythm Pharmaceuticals announced that the French National Agency for Medicines and Health Products Safety and French National Authority for Health have granted pre-marketing early access authorization AP1, for IMCIVREE. an MC4R agonist, for patients with lesional hypothalamic obesity. AP1 allows for early access to innovative therapies in France prior to European regulatory approval when a positive benefit/risk ratio is recognized and when no other therapeutic alternatives are available. The AP1 for setmelanotide was granted following review of efficacy and safety data from Rhythm’s Phase 2 trial by the ANSM and HAS. Products included in the AP1 programs are fully covered by France’s National Health System and Rhythm can expect to be reimbursed for any patients receiving treatments through this program. Setmelanotide has been available through a similar program for patients in France with Bardet-Biedl syndrome. Hypothalamic obesity is a rare, acquired form of extreme obesity that occurs following damage to the hypothalamic region of the brain, which includes the MC4R pathway and is responsible for controlling physiological functions such as hunger and weight regulation. It most frequently follows the growth or surgical removal of craniopharyngioma, astrocytoma or other rare brain tumors. Patients experience rapid weight gain, a reduction in energy expenditure and increase in hunger in the first six to 12 months following tumor resection, and ultimately develop severe obesity. Rhythm estimates there are approximately 500-2,000 patients living with hypothalamic obesity in France, with approximately 50 to 200 new cases each year. In the United States, the Company estimates the prevalence in hypothalamic obesity to be approximately 5,000-10,000 patients with an annual incidence of approximately 500 new patients. As previously announced, Rhythm’s Phase 3 trial in hypothalamic obesity is ongoing following strong Phase 2 trial data that showed meaningful weight loss was sustained and progressed in patients with hypothalamic obesity treated with setmelanotide. The Company enrolled 18 patients with hypothalamic obesity in its Phase 2 trial. Sixteen of 18 patients achieved the primary endpoint of 5 percent or greater reduction in body mass index after 16 weeks of treatment. Overall, there was a 14.5 mean percent reduction in BMI at Week 16 from baseline.