Rhythm Pharmaceuticals' Setmelanotide NDA Accepted by FDA for Acquired Hypothalamic Obesity Treatment

Rhythm Pharmaceuticals (RYTM) announced that the FDA has accepted its supplemental New Drug Application for setmelanotide to treat acquired hypothalamic obesity, with Priority Review granted. Analyst Michael Ulz from Morgan Stanley reiterated a Buy rating with a price target of $109, citing positive Phase 3 data and a good safety profile. The company is developing precision medicines for rare genetic obesity disorders.

Rhythm Pharmaceuticals (RYTM) has experienced a significant uptick in its stock price following the FDA's acceptance of its supplemental New Drug Application (sNDA) for setmelanotide to treat acquired hypothalamic obesity. The company received Priority Review from the FDA, with a decision date set for December 20, 2025.

Analyst Michael Ulz from Morgan Stanley reiterated a Buy rating on RYTM, setting a price target of $109. Ulz cited the positive Phase 3 data and the drug's good safety profile as the reasons behind the rating. The company is focused on developing precision medicines for rare genetic obesity disorders.

Setmelanotide, previously approved as IMCIVREE, demonstrated statistically significant and clinically meaningful results in the pivotal Phase 3 TRANSCEND trial. The study involved 120 patients with acquired hypothalamic obesity and achieved a placebo-adjusted reduction in body mass index (BMI) of -19.8% over 52 weeks. The drug was generally well-tolerated, with common adverse events including nausea, vomiting, diarrhea, and injection site reactions.

The approval of setmelanotide for acquired hypothalamic obesity marks a significant advancement in the treatment of this rare condition. Rhythm Pharmaceuticals estimates that there are 5,000 to 10,000 people living with acquired hypothalamic obesity in the U.S. and 3,500 to 10,000 in the EU.

Analysts have responded to the news with mixed signals. Jonathan Wolleben of Citizens JMP reiterated a Buy rating on RYTM, setting a price target of $135.00. Michael Ulz of Morgan Stanley also issued a Buy rating, while TR | OpenAI reiterated a Hold rating. The company’s market capitalization stands at $6.46 billion, with a P/E ratio of -32.70.

Rhythm Pharmaceuticals will host a “Commercial Readiness for Acquired Hypothalamic Obesity” event for investors and analysts on September 24, 2025, in Boston, MA. The event will feature insights from leading physicians and provide an overview of U.S. launch plans. A live webcast will be available under "Events and Presentations" in the Investor Relations section of the Rhythm Pharmaceuticals website.

References:
[1] https://www.nasdaq.com/articles/rhythm-pharmas-stock-tops-100-fda-decision-imcivree-label-expansion-due-december
[2] https://simplywall.st/stocks/us/pharmaceuticals-biotech/nasdaq-rytm/rhythm-pharmaceuticals/news/why-rhythm-pharmaceuticals-rytm-is-up-58-after-fda-grants-pr
[3] https://www.ainvest.com/news/citizens-jmp-analyst-boosts-rhythm-pharmaceuticals-buy-rating-135-price-target-2508/