Prader-Willi Syndrome

11
Pipeline Programs
5
Companies
12
Clinical Trials
1 recruiting
1
Approved Products

Pipeline by Development Stage

Preclinical
Phase 1
Phase 1/2
Phase 2
Phase 2/3
Phase 3
On Market
0
0
0
4
0
6
1
Early DiscoveryClinical DevelopmentMarket

Drug Modality Breakdown

Peptide
2100%
+ 10 programs with unclassified modality

On Market (1)

Approved therapies currently available

U
PITOLISANTApproved
pitolisant
Unknown Company
film coated

Competitive Landscape

5 companies ranked by most advanced pipeline stage

Soleno Therapeutics
Soleno TherapeuticsREDWOOD CITY, CA
5 programs
4
DCCRPhase 31 trial
DCCRPhase 31 trial
DCCRPhase 31 trial
DCCRPhase 31 trial
VYKAT XRN/A1 trial
Active Trials
NCT07450664Enrolling By Invitation200Est. Mar 2028
NCT03714373Completed115Est. Aug 2023
NCT03440814Completed127Est. May 2020
+2 more trials
Harmony Biosciences
Harmony BiosciencesPLYMOUTH MEETING, PA
3 programs
1
2
PitolisantPhase 31 trial
Pitolisant tabletPhase 31 trial
Pitolisant oral tabletsPhase 21 trial
Active Trials
NCT04257929Active Not Recruiting65Est. Sep 2028
NCT07219485Enrolling By Invitation150Est. Aug 2030
NCT06366464Recruiting134Est. Jul 2027
Rhythm Pharmaceuticals
2 programs
2
SetmelanotidePhase 2Peptide1 trial
SetmelanotidePhase 2Peptide1 trial
Active Trials
NCT02311673Completed40Est. Oct 2016
NCT06772597Active Not Recruiting18Est. Oct 2027
Gedeon Richter
Gedeon RichterHungary - Budapest
1 program
1
RGH-706Phase 21 trial
Active Trials
NCT05322096Completed39Est. Apr 2024
Novo Nordisk
Novo NordiskBAGSVAERD DENMARK, Denmark
1 program
Norditropin SimpleXxN/A1 trial
Active Trials
NCT00372125Completed46Est. Mar 2010

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
2027
2028
2029
2030
Harmony BiosciencesPitolisant
Harmony BiosciencesPitolisant tablet
Soleno TherapeuticsDCCR
Soleno TherapeuticsDCCR
Soleno TherapeuticsDCCR
Soleno TherapeuticsDCCR
Rhythm PharmaceuticalsSetmelanotide
Gedeon RichterRGH-706
Harmony BiosciencesPitolisant oral tablets
Rhythm PharmaceuticalsSetmelanotide
Soleno TherapeuticsVYKAT XR
Novo NordiskNorditropin SimpleXx

Clinical Trials (12)

Total enrollment: 1,017 patients across 12 trials

NCT07219485Harmony BiosciencesPitolisant

A Study of Pitolisant in Participants With Prader-Willi Syndrome

Start: Aug 2025Est. completion: Aug 2030150 patients
Phase 3Enrolling By Invitation
NCT06366464Harmony BiosciencesPitolisant tablet

A Study of Pitolisant in Patients With Prader-Willi Syndrome

Start: May 2024Est. completion: Jul 2027134 patients
Phase 3Recruiting
NCT05701774Soleno TherapeuticsDCCR

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

Start: Jan 2023Est. completion: Jun 202883 patients
Phase 3Active Not Recruiting
NCT04086810Soleno TherapeuticsDCCR

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

Start: Oct 2019Est. completion: Oct 20190
Phase 3Withdrawn
NCT03714373Soleno TherapeuticsDCCR

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
NCT03440814Soleno TherapeuticsDCCR

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

Start: May 2018Est. completion: May 2020127 patients
Phase 3Completed
NCT06772597Rhythm PharmaceuticalsSetmelanotide

A Study of Setmelanotide in Patients With Prader-Willi Syndrome

Start: Mar 2025Est. completion: Oct 202718 patients
Phase 2Active Not Recruiting
NCT05322096Gedeon RichterRGH-706

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

Start: Sep 2022Est. completion: Apr 202439 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
NCT02311673Rhythm PharmaceuticalsSetmelanotide

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
NCT07450664Soleno TherapeuticsVYKAT XR

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

Start: Nov 2025Est. completion: Mar 2028200 patients
N/AEnrolling By Invitation
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

Phase Legend

Preclinical— Lab & animal studies
Phase 1— Safety & dosing
Phase 2— Efficacy testing
Phase 3— Large-scale trials
On Market— Approved & available

Key Insights

6 late-stage (Phase 3) programs — potential near-term approvals
1 actively recruiting trials targeting 1,017 patients
5 companies competing in this space