FDA Approves Imcivree for Acquired Hypothalamic Obesity

By Chris Stiles • Friday, March 20 14:48 • Permanent Link

Rhythm Pharmaceuticals, Inc. has announced the FDA approval of an expanded indication for Imcivree® (setmelanotide) to treat patients with acquired hypothalamic obesity (HO). This rare disease is characterized by accelerated and sustained weight gain caused by an injury to the hypothalamus or hypothalamic dysfunction. The approval makes Imcivree the first and only FDA-approved therapy for acquired HO in adults and pediatric patients aged 4 years and older.

The approval was based on the results of the pivotal phase 3 transcend trial, which demonstrated a statistically significant -18.4% placebo-adjusted reduction in body mass index (BMI) in patients with acquired HO. Specifically, participants on setmelanotide therapy achieved a -15.8% reduction in BMI compared to a +2.6% increase among patients on placebo at 52 weeks.

In addition to its efficacy in acquired HO, Imcivree is also approved for adult and pediatric patients aged 2 years and older with syndromic or monogenic obesity due to Bardet-Biedl syndrome (BBS) or pro-opiomelanocortin (POMC), proprotein convertase subtilisin/kexin type 1 (PCSK1), or leptin receptor (LEPR) deficiency.

The company estimates that there are approximately 10,000 people living with acquired HO in the U.S. Imcivree is now available for patients in the U.S. immediately, and the company is committed to supporting access for patients to its medicines through its Rhythm inTune program, which provides personalized, ongoing educational support for individuals living with certain rare forms of obesity.

The announcement has been met with enthusiasm from patient advocacy groups, with the founder of the Raymond A. Wood Foundation expressing hope and a path forward for thousands of patients who have long been without treatment options.

Imcivree's approval represents a transformative milestone for Rhythm Pharmaceuticals, reinforcing the company's commitment to bringing meaningful therapies to patients living with rare neuroendocrine diseases. Following these announcements, the company's shares moved -4.18%, and are now trading at a price of $101.63. Check out the company's full 8-K submission here.

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