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    PWS Therapeutics in Development

    Explore drugs, devices, and other approaches being developed to address the complex symptoms of Prader-Willi syndrome, from early research through clinical trials.

    Active Clinical Trials

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    Support PWS Research

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    with a request body that specifies how to map the columns of your import file to the associated CRM properties in HubSpot.... In the request JSON, define the import file details, including mapping the spreadsheet's columns to HubSpot data. Your request JSON should include the following fields:... entry for each column.

    Explore the PWS Treatment Pipeline

    This overview tracks drugs, devices, and other therapeutic approaches currently being studied for PWS. Projects range from early discovery and preclinical research to Phase 1, 2, and 3 clinical trials. It also includes existing FDA-approved drugs and devices currently being evaluated specifically in people with PWS.

    Use the categories below to explore current approaches. For additional study details and enrollment information, visit our active clinical trials directory.

    Many of these projects and programs have been made possible by FPWR fundraisers and donors who help move this promising research forward.

    Genetic Therapies

    Genetic Therapies – These therapies seek to activate the PWS region genes on the silent maternal chromosome 15 OR provide critical missing genes OR provide a substitute function for PWS genes. These are early stage projects – optimizing approaches and examining feasibility / efficacy in cell and animal models of PWS. Learn more about genetic therapy for PWS >>

    Gene Activation by Small Molecules Discovery / Preclinical View details Hide details

    How It Works

    Drugs targeting enzymes that establish/maintain epigenetic marks, activating the PWS genes on the maternal chromosome 15.

    Investigators / Companies

    Dr. Jiang

    FPWR Funded

    Dr. Blewitt

    FPWR Funded

    Advantages

    Addresses the underlying cause of PWS, so has the potential to profoundly impact symptoms. Would be applicable to all subtypes of PWS (deletion, UPD, imprinting).

    Potential Limitations

    Could activate other genes within or outside the PWS region; those consequences are not yet known. There may be critical “windows" in which the therapy would be most effective.
    CRISPR-Based Activation Discovery View details Hide details

    How It Works

    Delivery of a specialized gene/protein to specifically target and activate the PWS region on the normally silent maternal chromosome 15.

    Investigators / Companies

    Dr. Gersbach
    FPWR Funded
    Dr. Lu
    FPWR Funded

    Advantages

    Addresses the underlying cause of PWS, so has the potential to profoundly impact symptoms. Applicable to all subtypes. Specificity may be better.  Potential for a therapy that only has to be delivered once. 

    Potential Limitations

    Gene delivery is likely to be challenging. Gene therapy is new, so the FDA approval path may be difficult. Potential for ‘off-target’ effects.
    Oligonucleotide Therapy Preclinical View details Hide details

    How It Works

    Use of small pieces DNA or RNA to modify gene expression.

    Investigators / Companies

    Dr. Lee
    FPWR Funded

    Advantages

    Addresses the underlying cause of PWS, so has the potential to profoundly impact symptoms. Dosage may be better titrated than other gene therapy approaches. Delivery may be more readily accomplished.

    Potential Limitations

    Need for repeat therapy. Oligonucleotide must be brain-penetrant and will probably need to target a large number of cells – this will be challenging.
    Epigenome Editing Discovery View details Hide details

    How It Works

    Designer epigenome modifiers.

    Investigators / Companies

    Dr. Mussolino
    FPWR Funded

    Advantages

    Addresses the underlying cause of PWS, has the potential to profoundly impact symptoms. Applicable to all subtypes.

    Potential Limitations

    Development and delivery of epigenetic modifiers are at an early stage. Targeting and delivery will be challenging.

    Hyperphagia, Behavior, and Appetite

    Hyperphagia, Appetite, and Behavior:  These drugs are being evaluated for their ability to curb appetite and/or induce weight loss and may favorably impact behavior / social interaction. See our Clinical Trials Directory for more information on Active Clinical Trials (Phase 1, 2, and 3) for people with PWS. 
    PDE10 Inhibitor Phase 3 planned for 2026. View details Hide details

    How It Works

    PBF-999 is a PDE10 inhibitor believed to modulate central appetite regulation.

    Investigators / Companies

    Palobiofarma
    MC4R Agonist Phase 2 View details Hide details

    How It Works

    Works the MC4R pathway, which is important for appetite regulation. 

    Investigators / Companies

    Rhythm
    Triple Monamine Reuptake Inhibitor Phase 2 View details Hide details

    How It Works

    CSTI-500 is a triple reuptake inhibitor, modulating serotonin, dopamine, and norepinephrine.

    Investigators / Companies

    Consynance
    GLP Receptor Agonists FDA approved, pilot studies in PWS View details Hide details

    How It Works

    Suppresses appetite.

    Investigators / Companies

    Various
    Semaglutide
    Tirzepatide
    Retatrutide

    Advantages

    FDA approved. May improve glucose metabolism and increase satiety.

    Potential Limitations

    May slow gut motility, which can be problematic in PWS. Weight loss in PWS has not yet been assessed.
    Oxytocin Phase 2 & 3 View details Hide details

    How It Works

    Binds to oxytocin receptors, regulates trust, emotions, and appetite. 

    Investigators / Companies

    Tonix Pharmaceuticals
    OT4B

    Daytime Sleepiness

    Individuals with PWS often struggle with daytime sleepiness, which can interfere with school, work, and social activities. Researchers are evaluating these drugs for their ability to reduce daytime sleepiness. See our Clinical Trials Directory for more information on Active Clinical Trials (Phase 1, 2, and 3) for people with PWS.
    Pitolisant Phase 3 View details Hide details

    How It Works

    Pitolisant blocks histamine-3 receptors in the brain to help you stay awake and alert without acting as a traditional stimulant.

    Investigators / Companies

    Harmony Biosciences

    Advantages

    Approved for Narcolepsy in the US and the EU. Potential to also impact behavior and cognition.

    Potential Limitations

    Not yet FDA-approved.

    Procedures / Devices

    Procedures / Devices – these devices may improve appetite and behavior in PWS
    Cerebellar TMS Pilot View details Hide details

    How It Works

    Magnetic stimulation of the part of the cerebellum that may be important in appetite regulation.

    Investigators / Companies

    Dr Holsen

    Advantages

    Noninvasive, non drug intervention

    Potential Limitations

    Safety and efficacy haven't been established yet.
    Targeted Ultrasound Preclinical View details Hide details

    How It Works

    Non-invasive ultrasound can be used to stimulate the secretion of hormones that are deficient in PWS. 

    Investigators / Companies

    Dr. Puleo
    FPWR Funded
    Dr. Whitcomb
    FPWR Funded

    Advantages

     Non-invasive. 

    Potential Limitations

    Not yet tested in humans.
    Vagus Nerve Stimulation Phase 3 Enrollment Complete View details Hide details

    How It Works

    Vagus nerve may not communicate effectively with the brain in PWS.

    Investigators / Companies

    FPWR

    Advantages

    Approved for some mental health problems and epilepsy. New devices are noninvasive. Degree of stimulation can be tightly regulated by the device.

    Potential Limitations

    Initial studies encouraging, but have been small to date.