Obesity due to prohormone convertase I deficiency

MONDO:0010961

Prohormone convertase-I deficiency is the rarest form of monogenic obesity. The disorder is characterized by severe childhood obesity, hypoadrenalism, reactive hypoglycaemia, and elevated circulating levels of certain prohormones.

Also known as: PCI deficiency, PCSK1 Deficiency, obesity and endocrinopathy due to impaired processing of prohormones, proprotein convertase 1/3 deficiency

81 clinical trials for this condition and its sub-types, 1 tagged with Obesity due to prohormone convertase I deficiency itself.

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Where it sits in the disease tree

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Part of

↑ Congenital hypogonadotropic hypogonadism (83) ↑ Inherited obesity (9)

Including sub-types (81) Tagged with Obesity due to prohormone convertase I deficiency (1)

Completed 1

Gene-Targeted obesity drug shows promise in phase 3 trial

Disease control Completed

This phase 3 trial is testing a drug called setmelanotide in people with obesity linked to certain gene variants (POMC, PCSK1, LEPR, NCOA1, or SH2B1). About 296 participants, aged 6 to 65, will receive either the drug or a placebo to see if it helps reduce body weight. The study …

Phase 3 • Sponsor: Rhythm Pharmaceuticals, Inc. • Aim: Disease control

Last updated Aug 08, 2026 00:03 UTC

Obesity due to prohormone convertase I deficiency

Where it sits in the disease tree

Gene-Targeted obesity drug shows promise in phase 3 trial