Rhythm Pharmaceuticals Announces Pre-Marketing Early Access Authorization for Setmelanotide for Use in Patients with Hypothalamic Obesity in France

-- Reimbursed early access program allows for patients with hypothalamic obesity in France to receive setmelanotide treatment --
-- Early access designation for hypothalamic obesity comes in addition to Bardet-Biedl syndrome --

BOSTON, Aug. 07, 2023 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (Nasdaq: RYTM), a commercial-stage biopharmaceutical company focused on transforming the lives of patients and their families living with hyperphagia and severe obesity caused by rare melanocortin-4 receptor (MC4R) pathway diseases, today announced that the French National Agency for Medicines and Health Products Safety (ANSM) and French National Authority for Health (HAS) have granted pre-marketing early access authorization AP1 (Autorisation d’Accès Précoce), for IMCIVREE (setmelanotide), 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 (BBS) since July 2022.

“We are delighted to announce that the French regulatory authorities granted this unique AP1 status to setmelanotide, as this is a recognition of the seriousness of the unmet medical need and what we believe to be a significant first step towards our goal of expanding access to setmelanotide to include patients with hypothalamic obesity globally,” said Yann Mazabraud, Executive Vice President and Head of International at Rhythm. “We look forward to continuing to work with French experts to deliver setmelanotide to patients in France.”

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.