This is a list of investigational Prader–Willi syndrome drugs, or drugs that are currently under development for clinical use for the treatment of Prader–Willi syndrome (PWS) but are not yet approved. Chemical/generic names are listed first, with developmental code names, synonyms, and brand names in parentheses. The format of list items is "Name (Synonyms) – Mechanism of Action [Reference]". This list was last comprehensively updated in July 2026. It is likely to become outdated with time.
Under development
Phase 3 Denatonium acetate (ARD-101) – taste receptor 2 (TAS2R; bitter taste receptor) agonist [1] Pitolisant (BF-2.649; BF-2649; HBS-201; HBS-301; Ozawade; tiprolisant; Wakix) – histamine H3 receptor antagonist [2]
Phase 2/3 Cannabidiol synthetic (CBD; pharmaceutical cannabidiol; RAD-011) – non-psychoactive cannabinoid and various actions [3]
Phase 2 BMB-101 – serotonin 5-HT2C receptor agonist [4] Cannabidivarin (CBDV; GWP-42006) – non-psychoactive cannabinoid and various actions [5] CSTI-500 (AMR-001181) – serotonin–norepinephrine–dopamine reuptake inhibitor (SNDRI) [6] Ercanetide (NNZ-2591; ACP-2591; cyclo-L-glycyl-L-2-allylproline; PMS-001) – synthetic analogue of cyclic glycine-proline (cGP; traneurocin) and various actions [7] GDD-3898 – stearoyl-CoA desaturase (SCD) inhibitor [8] RGH-706 – melanin-concentrating hormone receptor 1 (MCHR1) antagonist [9] Sepranolone (isoallopregnanolone; epiallopregnanolone; UC-1010) – GABAA receptor negative allosteric modulator and neurosteroid [10] Setmelanotide (BIM-22493; CAM-4072; Imcivree; RM-493) – melanocortin MC4 receptor agonist [11] Tesofensine/metoprolol (metoprolol/tesofensine; Tesomet) – combination of tesofensine (serotonin–norepinephrine–dopamine reuptake inhibitor (SNDRI)) and metoprolol (beta blocker) [12]
Phase 1/2 RM-718 – melanocortin MC4 receptor agonist [13]
Phase 1 BMB-105 – serotonin 5-HT2C receptor agonist [14] Oxytocin (intranasal potentiated oxytocin; oxytocin intranasal; TI-001; TI-114; TNX-1900; TNX-2900) – oxytocin receptor agonist [15] [16]
Preclinical HBS-102 (CSTI-100) – melanin-concentrating hormone receptor 1 (MCHR1) antagonist [17]
Research NP-1031 – oxytocin receptor modulator [18]
Phase unknown BSN-272 – undefined mechanism of action [19]
Not under development
No development reported GLWL-01 - ghrelin O-acyltransferase (GOAT) inhibitor [20] HM-04 – ghrelin receptor antagonist [21] RM-853 (T-3525770) – ghrelin O-acyltransferase (GOAT) inhibitor [22]
Discontinued Beloranib (6-O-(4-dimethylaminoethoxy) cinnamoyl fumagillol hemioxalate; beloranib hemioxalate; CKD-732; TNP-470 analogue; ZGN-433; ZGN-440) – methionine aminopeptidase 2 (METAP2) inhibitor [23] Carbetocin intranasal (ACP-101; FE-992097; LV-101) – oxytocin receptor agonist [24] [25] Livoletide (AZP-531; UAG-analogue-Alize; unacylated-analogue-Alize) – unacylated ghrelin (UAG) analogue and acylated ghrelin antagonist [26] NM-136 (LOB-0136) – monoclonal antibody against gastric inhibitory polypeptide (GIP) [27] Zevaquenabant (INV-101) – cannabinoid CB1 receptor antagonist [28]
Clinically used drugs
Approved drugs Diazoxide choline controlled release (C601; C602; DCCR; Vykat XR) – KATP potassium channel opener [29] Somatropin (Genotonorm; Genotropin; PN-180,307) – human growth hormone (hGH) replacement [30] Somatropin (Humatrope; Hutrope; LY-137998; Umatrope) – human growth hormone (hGH) replacement [31] Somatropin biosimilar (Omnitrope; PrOmnitrope) – human growth hormone (hGH) replacement [32]
See also Lists of investigational drugs
References
External links AdisInsight - Springer