PWSA | USA received news from Harmony Biosciences that the company is working on a plan to begin their Phase 3 clinical trial for pitolisant, also known as WAKIX, following a positive End-of-Phase 2 meeting with the U.S. Food and Drug Administration. WAKIX is an FDA approved drug used to treat excessive daytime sleepiness or cataplexy in adult patients with narcolepsy.
According to Harmony Biosciences press release announcement, Chief Medical Officer Kumar Budur, MD shared, "We are pleased with the outcome of our End-of-Phase 2 meeting with the FDA as we prepare to initiate our Phase 3 registrational study, which aims to further investigate the efficacy and safety of pitolisant as a potential treatment for excessive daytime sleepiness in individuals with Prader-Willi syndrome. Building upon the encouraging data obtained from our Phase 2 signal detection study, we remain committed to advancing our development program for pitolisant in pursuit of a new indication in people with PWS, given the high unmet medical need in this population.”
Find Harmony Biosciences' full press release announcement at the button below, which includes more details from their meeting with the FDA and next steps for the Phase 3 study.
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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.