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Prader-Willi Syndrome

9
Pipeline Programs
18
Companies
25
Clinical Trials
2 recruiting
0
Approved Products

Pipeline by Development Stage

Preclinical
Phase 1
Phase 1/2
Phase 2
Phase 2/3
Phase 3
On Market
0
1
0
5
0
2
1
Early DiscoveryClinical DevelopmentMarket

Drug Modality Breakdown

Peptide
5100%
+ 21 programs with unclassified modality

Competitive Landscape

17 companies ranked by most advanced pipeline stage

Grace Medical
Grace MedicalTN - Memphis
1 program
1
TirzepatidePhase 4Peptide1 trial
Active Trials
NCT06901245Recruiting36Est. Dec 2026
UNION therapeutics
UNION therapeuticsDenmark - Hellerup
1 program
1
Recombinant Human Growth HormonePhase 31 trial
Active Trials
NCT03554031Unknown30Est. Jan 2020
Pfizer
PfizerNEW YORK, NY
1 program
1
somatropin - GH naïve pediatric cohortPhase 31 trial
Active Trials
NCT04697381Completed33Est. Apr 2024
ConSynance Therapeutics
2 programs
1
1
CSTI-500Phase 21 trial
CSTI-500Phase 11 trial
Active Trials
NCT05504395Completed10Est. Feb 2023
NCT07348601Not Yet Recruiting12Est. Jun 2027
Colorado Therapeutics
Colorado TherapeuticsCO - Louisville
1 program
1
ARD-101Phase 21 trial
Active Trials
NCT05153434Completed19Est. Sep 2024
Aardvark Therapeutics
Aardvark TherapeuticsCA - San Diego
1 program
1
ARD-101Phase 2
Bright Minds Biosciences
1
BMB-101Phase 21 trial
Active Trials
NCT07266324Not Yet Recruiting16Est. Mar 2027
Neuren Pharmaceuticals
Neuren PharmaceuticalsAustralia - Camberwell
1 program
1
NNZ-2591Phase 21 trial
Active Trials
NCT05879614Withdrawn0Est. Jun 2025
Soleno Therapeutics
Soleno TherapeuticsREDWOOD CITY, CA
5 programs
VYKAT XRN/A1 trial
DCCRPHASE_31 trial
DCCRPHASE_31 trial
DCCRPHASE_31 trial
DCCRPHASE_31 trial
Active Trials
NCT07450664Enrolling By Invitation200Est. Mar 2028
NCT03440814Completed127Est. May 2020
NCT05701774Active Not Recruiting83Est. Jun 2028
+2 more trials
Harmony Biosciences
Harmony BiosciencesPLYMOUTH MEETING, PA
3 programs
Pitolisant oral tabletsPHASE_21 trial
PitolisantPHASE_31 trial
Pitolisant tabletPHASE_31 trial
Active Trials
NCT04257929Active Not Recruiting65Est. Sep 2028
NCT07219485Enrolling By Invitation150Est. Aug 2030
NCT06366464Recruiting134Est. Jul 2027
Human BioSciences
Human BioSciencesWV - Martinsburg
2 programs
Group 1N/A1 trial
TopiramateN/A1 trial
Active Trials
NCT00375089Completed392Est. Jan 2014
NCT00065923Completed10
Rhythm Pharmaceuticals
2 programs
SetmelanotidePHASE_2Peptide1 trial
SetmelanotidePHASE_2Peptide1 trial
Active Trials
NCT06772597Active Not Recruiting18Est. Oct 2027
NCT02311673Completed40Est. Oct 2016
Angeles Therapeutics
Angeles TherapeuticsCA - Los Angeles
1 program
ExenatideN/APeptide1 trial
Active Trials
NCT01444898Completed10Est. Dec 2013
Novo Nordisk
Novo NordiskBAGSVAERD DENMARK, Denmark
1 program
Norditropin SimpleXxN/A1 trial
Active Trials
NCT00372125Completed46Est. Mar 2010
Sandoz
SandozAustria - Kundl
1 program
OctreotideN/APeptide1 trial
Active Trials
NCT00399893Terminated5Est. Sep 2010
Integrated Biosciences
Integrated BiosciencesCA - San Carlos
1 program
Safe and Sound ProtocolN/A1 trial
Active Trials
NCT03871751Terminated10Est. Dec 2019
Gedeon Richter
Gedeon RichterHungary - Budapest
1 program
RGH-706PHASE_21 trial
Active Trials
NCT05322096Completed39Est. Apr 2024

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
2027
2028
2029
2030
Grace MedicalTirzepatide
Harmony BiosciencesPitolisant
Harmony BiosciencesPitolisant tablet
Soleno TherapeuticsDCCR
Pfizersomatropin - GH naïve pediatric cohort
Soleno TherapeuticsDCCR
Soleno TherapeuticsDCCR
Soleno TherapeuticsDCCR
UNION therapeuticsRecombinant Human Growth Hormone
ConSynance TherapeuticsCSTI-500
Bright Minds BiosciencesBMB-101
Rhythm PharmaceuticalsSetmelanotide
Neuren PharmaceuticalsNNZ-2591
Gedeon RichterRGH-706
Colorado TherapeuticsARD-101

Showing 15 of 25 trials with date data

Clinical Trials (25)

Total enrollment: 1,600 patients across 25 trials

Tirzepatide in PWS, HO and GNSO

Start: May 2025Est. completion: Dec 202636 patients
Phase 4Recruiting

A Study of Pitolisant in Participants With Prader-Willi Syndrome

Start: Aug 2025Est. completion: Aug 2030150 patients
Phase 3Enrolling By Invitation

A Study of Pitolisant in Patients With Prader-Willi Syndrome

Start: May 2024Est. completion: Jul 2027134 patients
Phase 3Recruiting

Open-Label Extension Study of DCCR in Patients With Prader-Willi Syndrome

Start: Jan 2023Est. completion: Jun 202883 patients
Phase 3Active Not Recruiting
NCT04697381Pfizersomatropin - GH naïve pediatric cohort

Study of the Efficacy and Safety of Somatropin in Japanese Participants With PWS

Start: Feb 2021Est. completion: Apr 202433 patients
Phase 3Completed

An Open-Label Study of DCCR Tablet in Patients With PWS

Start: Oct 2019Est. completion: Oct 20190
Phase 3Withdrawn

Open-Label Extension Study of DCCR in PWS Followed by Double-Blind, Placebo-Controlled, Randomized Withdrawal Period

Start: Oct 2018Est. completion: Aug 2023115 patients
Phase 3Completed

A Study of Diazoxide Choline in Patients With Prader-Willi Syndrome

Start: May 2018Est. completion: May 2020127 patients
Phase 3Completed
NCT03554031UNION therapeuticsRecombinant Human Growth Hormone

A Study to Evaluate the Efficacy and Safety of Recombinant Human Growth Hormone Injection in Patients With Prader-Willi Syndrome

Start: Apr 2018Est. completion: Jan 202030 patients
Phase 3Unknown

A Study of CSTI-500 in Patients With Prader-Willi Syndrome

Start: Mar 2026Est. completion: Jun 202712 patients
Phase 2Not Yet Recruiting

A 2-Part Study to Assess Efficacy, Safety and Tolerability of BMB-101 for the Treatment of Patients With Prader-Willi Syndrome.

Start: Jan 2026Est. completion: Mar 202716 patients
Phase 2Not Yet Recruiting

A Study of Setmelanotide in Patients With Prader-Willi Syndrome

Start: Mar 2025Est. completion: Oct 202718 patients
Phase 2Active Not Recruiting

An Open-Label Study of Oral NNZ-2591 in Prader-Willi Syndrome (PWS-001)

Start: Sep 2023Est. completion: Jun 20250
Phase 2Withdrawn

Study to Evaluate Efficacy, Safety, and Tolerability of RGH-706 in Prader-Willi Syndrome

Start: Sep 2022Est. completion: Apr 202439 patients
Phase 2Completed

A Study of Oral ARD-101 in Patients With Prader-Willi Syndrome

Start: May 2022Est. completion: Sep 202419 patients
Phase 2Completed
NCT04257929Harmony BiosciencesPitolisant oral tablets

A Phase 2 Study to Evaluate the Safety and Efficacy of Pitolisant in Patients With Prader-Willi Syndrome, Followed by an Open Label Extension

Start: Dec 2020Est. completion: Sep 202865 patients
Phase 2Active Not Recruiting

Phase 2 Trial to Evaluate Safety and Efficacy of Setmelanotide (RM-493) in Obese Participants With Prader-Willi Syndrome

Start: Mar 2015Est. completion: Oct 201640 patients
Phase 2Completed

A Pharmacokinetic and Safety Study of CSTI-500 in Subjects With Prader-Willi Syndrome

Start: Nov 2022Est. completion: Feb 202310 patients
Phase 1Completed

Observational Study of VYKAT™ XR in Patients With Prader-Willi Syndrome

Start: Nov 2025Est. completion: Mar 2028200 patients
N/AEnrolling By Invitation
NCT03871751Integrated BiosciencesSafe and Sound Protocol

Home-based SSP on Individuals With PWS

Start: Apr 2019Est. completion: Dec 201910 patients
N/ATerminated

Effects of Exenatide on Overweight Adolescents With Prader-Willi Syndrome

Start: Mar 2012Est. completion: Dec 201310 patients
N/ACompleted

Octreotide Therapy in Children and Young Adults With Prader-Willi Syndrome (PWS)

Start: Dec 2006Est. completion: Sep 20105 patients
N/ATerminated

Characteristics of Prader-Willi Syndrome and Early-onset Morbid Obesity

Start: Sep 2006Est. completion: Jan 2014392 patients
N/ACompleted
NCT00372125Novo NordiskNorditropin SimpleXx

Nordic Study on the Effects of Growth Hormone (Norditropin SimpleXx) Treatment in Adults With Prader-Willi Syndrome

Start: Apr 2005Est. completion: Mar 201046 patients
N/ACompleted

Treatment of Self-Injurious Behavior in Individuals With Prader-Willi Syndrome

Start: Jul 200210 patients
N/ACompleted

Related Jobs

Phase Legend

PreclinicalLab & animal studies
Phase 1Safety & dosing
Phase 2Efficacy testing
Phase 3Large-scale trials
On MarketApproved & available

Key Insights

2 late-stage (Phase 3) programs, potential near-term approvals
2 actively recruiting trials targeting 1,600 patients
18 companies competing in this space

The information on this page is for informational purposes only and should not be used as a substitute for professional medical advice. Drug information is sourced from FDA, DailyMed, and other government databases. Adverse event data from FAERS does not establish causation. Always consult a healthcare professional for medical decisions.