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OTBB1 (along with its related clinical programs OTBB2 and OTBB3) is an intranasal formulation of the peptide hormone oxytocin, developed by the French biotechnology company OT4B in collaboration with the University Hospital of Toulouse (CHU Toulouse). The drug is specifically designed as an early therapeutic intervention for Prader-Willi Syndrome (PWS), a genetic disorder characterized by a deficiency in oxytocin-producing neurons in the hypothalamus. By administering oxytocin intranasally, the therapy acts as an agonist at the oxytocin receptor (OXTR) to compensate for this deficiency. The development program aims to improve feeding behaviors (such as sucking in neonates), social interaction, and behavioral disorders across different age groups, from the neonatal period through adulthood. OTBB1 has received orphan drug designation in both the United States and the European Union.
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