We are excited to share that Levo’s New Drug Application (NDA) for carbetocin as a treatment for PWS will be discussed at a public meeting of the Psychopharmacologic Drugs Advisory Committee to be held November 4, 2021. This is a major step forward on this drug's path through the approval process. The Food and Drug Administration (FDA) calls on Advisory Committees to provide independent advice that contributes to the agency's regulatory decision-making. Patients, healthcare providers, and other interested community members are encouraged to share their views with the Advisory Committee.
This is an exciting moment and is our chance for the PWS community to show our support of this potential new treatment. Here's some information and details about how we can work together to support this critical next step:
- The meeting will take place virtually through an online teleconference on November 4, 2021, from 10 a.m. to 4 p.m. Eastern Time.
- Electronic comments may be submitted on or before November 3, 2021. Visit https://www.regulations.gov to submit your comments [Docket No. FDA-2021-N-0860]. Comments received on or before October 21, 2021, will be provided to the Advisory Committee. Comments received after that date will be taken into consideration by FDA.
What is an Advisory Committee?
Advisory committees provide independent advice and recommendations to the Food and Drug Administration (FDA) on scientific and technical matters related to the development and evaluation of products regulated by the Agency. Although the committees provide recommendations to the Agency, final decisions are made by the FDA.
Advisory committee meetings can occur during any stage of a medical products review process and after a product has been approved and marketed. Typically, an advisory committee meeting is held to assist the review division with interpretation when questions or difficulties related to trial data arise.
What will happen during the Advisory Committee meeting?
During the public meeting, the company sponsor presents its data, the FDA scientists present their review, and members of the public are invited to speak briefly during the “open public comment period." Advisory Committee members ask questions of the sponsor, the FDA, and occasionally individuals who speak during the open public comment period. At most advisory committee meetings, most of the research-based presentations are by the company and its paid consultants, with less time for presentations by FDA scientists. Outside experts, such as government researchers or independently funded researchers, are sometimes invited to make formal presentations at an advisory committee meeting, but such presentations are not typical.
After reviewing the data and listening to the company presentation, the FDA presentation, and any public comments, committee members discuss and vote on questions that the FDA has prepared and provided to committee members in advance. The prepared questions for new medical products include whether the product is effective, whether it is safe, and “whether the safety and effectiveness information submitted for a new drug is adequate for marketing approval.”
Who can attend an Advisory Committee meeting?
Advisory Committee meetings are public and open to anyone who wishes to attend. This Advisory Committee meeting will be held via online teleconference.
How can you participate in the Advisory Committee meeting?
Community members may provide written comments or request an oral presentation.
- If you are a PWS community member but did not participate in the Carbetocin clinical trial, we encourage you to submit a written comment to the advisory committee.
- If your family has participated in the Carbetocin clinical trial, you may be interested in sharing your experience with the committee through a short, oral testimony.
Speaking time is limited (one hour). Depending on how many individuals sign up to speak, allotted times may range from two-five minutes. An interested person who wishes to make an oral presentation during the Open Public Hearing portion of an Advisory Committee meeting should register with FDA before the meeting. Note that because speaking time is so limited, we encourage community members who do not have direct experience with Carbetocin to submit written comments rather than request a speaking slot.
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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.