Rhythm Pharmaceuticals Inc., a company developing medicines for rare genetic disorders of obesity, has won FDA approval for its first product, Imcivree (setmelanotide), following a priority review. The drug is designed to restore a biological pathway that, when disrupted, can lead to constant hunger. The approval covers the treatment of three types of ultrarare early onset obesity tied to pro-opiomelanocortin (POMC), proprotein convertase subtilisin/kexin type 1 (PCSK1) and leptin receptor (LEPR) deficiencies, when confirmed by genetic testing.
In a Phase 2 trial of PWS subjects, setmelanotide failed to show a significant change in hyperphagia, therefore hyperphagia in PWS does not have an FDA indication for treatment with Imcivree. There may be instances where the drug is prescribed for individuals with PWS, but this would be an “off label” use of the medication. Expensive treatment with medications for rare diseases that are not indicated by the FDA would typically require physician justification to convince insurance companies to cover drug costs— and insurance companies might not agree to pay. There are safety considerations as well. When a medication has not completed the clinical trial process, there is incomplete safety data for the drug in certain populations. And the failed study implies that based on current data it is unlikely the drug would help in PWS.
PWSA | USA is supportive of all potential research opportunities in PWS and monitors scientific research. We advocate for our families by supporting current research efforts and working with researchers and companies actively pursuing research to identify treatments to improve the quality of life for individuals with PWS.
If you have further questions, please call Mary Burr, Medical and Research Coordinator, at 800-926-4797 or email her at mburr@pwsausa.org
Jennifer Bolander has been serving as a Special Education Specialist for PWSA (USA) since October of 2015. She is a graduate of John Carroll University and lives in Ohio with her husband Brad and daughters Kate (17), and Sophia (13) who was born with PWS.
Perry A. Zirkel has written more than 1,500 publications on various aspects of school law, with an emphasis on legal issues in special education. He writes a regular column for NAESP’s Principal magazine and NASP’s Communiqué newsletter, and he did so previously for Phi Delta Kappan and Teaching Exceptional Children.
Evan has worked with the Prader-Willi Syndrome Association (USA) since 2007 primarily as a Crisis Intervention and Family Support Counselor. Evans works with parents and schools to foster strong collaborative relationships and appropriate educational environments for students with PWS.
Dr. Amy McTighe is the PWS Program Manager and Inpatient Teacher at the Center for Prader-Willi Syndrome at the Children’s Institute of Pittsburgh. She graduated from Duquesne University receiving her Bachelor’s and Master’s degree in Education with a focus on elementary education, special education, and language arts.
Staci Zimmerman works for Prader-Willi Syndrome Association of Colorado as an Individualized Education Program (IEP) consultant. Staci collaborates with the PWS multi-disciplinary clinic at the Children’s Hospital in Denver supporting families and school districts around the United States with their child’s Individual Educational Plan.
Founded in 2001, SDLC is a non-profit legal services organization dedicated to protecting and advancing the legal rights of people with disabilities throughout the South. It partners with the Southern Poverty Law Center, Protection and Advocacy (P&A) programs, Legal Services Corporations (LSC) and disability organizations on major, systemic disability rights issues involving the Individuals with Disabilities Education Act (IDEA), Americans with Disabilities Act (ADA), and the federal Medicaid Act. Recently in November 2014, Jim retired.