Rhythm Pharmaceuticals received a three‑month extension from the U.S. Food and Drug Administration to review its supplemental New Drug Application for setmelanotide in acquired hypothalamic obesity, moving the PDUFA goal date from December 20 2025 to March 20 2026.
The extension follows the FDA’s request for additional sensitivity analyses of the Phase 3 TRANSCEND trial data. No new clinical, safety, or manufacturing data were requested, and the request was classified as a major amendment, giving Rhythm extra time to submit the analyses.
While the delay postpones the potential launch of setmelanotide for this rare indication, it also signals that the agency is not rejecting the application. The additional three months provide Rhythm with a buffer to strengthen the submission and could position the company for a faster approval once the analyses are completed.
Rhythm’s Q3 2025 financial results show net product revenue of $51.3 million, up 6 % sequentially and 54.3 % year‑over‑year, driven largely by sales of IMCIVREE for Bardet‑Biedl syndrome. The company posted a net loss of $54.3 million versus $45.0 million in Q3 2024, but its cash balance stood at $416.1 million as of September 30 2025, and gross profit margins remained high at 89.4 %.
CEO David Meeker said the company remains confident in the data and the drug’s potential, noting that the strong cash position and progress across the melanocortin‑4 receptor pipeline position Rhythm to deliver sustained, long‑term growth.
Setmelanotide is already approved for obesity caused by Bardet‑Biedl syndrome, POMC, PCSK1, and LEPR deficiencies, and the Phase 3 TRANSCEND trial met its primary endpoint with a 19.8 % placebo‑adjusted BMI reduction in patients with acquired hypothalamic obesity, a condition that currently has no FDA‑approved treatments.
The extension underscores the regulatory scrutiny Rhythm faces as it expands its product portfolio, but the company’s robust financial footing and the compelling clinical evidence for setmelanotide suggest that the delay is a procedural hurdle rather than a setback, and the company is positioning itself to capitalize on a significant new market opportunity once the review is complete.
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