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Moving Care of SMS Forward

By: Maggie Miller

On December 1, 2020, the Food and Drug Administration (FDA) approved the use of HETLIOZ® (tasimelteon) capsule and liquid formulations for the treatment of adults and children, respectively, with nighttime sleep disturbances associated with Smith-Magenis Syndrome (SMS). This is the first medication ever approved for use in persons with Smith-Magenis syndrome. VANDA has worked closely with the SMS community over several years to develop a clinical trial of the medication, tasimelteon, which progressed from an observational/interventional study to a PK (pharmacokinetic) study, to enrollment in the clinical trial of the medication.

PRISMS was first invited to a face-to-face meeting with VANDA Pharmaceuticals in May of 2013 where parents were allowed to share the everyday challenges that families face in regard to sleep issues for persons with SMS. Parents were also invited to provide helpful guidance in the design of the study to make the experience a bit easier for the participants and their caregivers.

The study continued with the expansion of trial sites across the U.S. and the extension of the trial to include in-home visits from a nurse to reduce the number of travel requirements for families and the stress related to travel. A few families were also able to speak directly to an FDA panel about the impact of the sleep challenges on their SMS child and the impact on the entire family.

VANDA understood the tremendous sacrifices that families would undertake in participating in these trials and thus, VANDA’s willingness to allow input and access from PRISMS, the Professional Advisory Board (PAB), and families projected that understanding. We are grateful for the ability to have been a small voice in the design and implementation of the study and hope this medication can make a difference in the lives of our SMS individuals and families.

“The FDA approval of HETLIOZ® for the treatment of nighttime sleep disturbances in SMS would not have been accomplished without the heroic efforts of SMS patients and the efforts of their families and advocates supporting the recruitment, design, and conduct of the study. We remain committed to providing this much-needed therapy to patients with SMS.”

Mihael H. Polymeropou- los M.D., Vanda’s President, and CEO

HETLIOZSolutions was created to support people who have been diagnosed with nighttime sleep disturbances in SMS. The program can assist with coordinating care to help answer insurance questions, as well as provide financial support and educational resources. For more information go to: https://