TREND Community Announces Call-to-Action Editorial on Prader-Willi Syndrome in the International Journal of Molecular Sciences
PHILADELPHIA, August 9, 2023 – TREND Community announces the publication of an editorial, “The Arduous Path to Drug Approval for the Management of Prader-Willi Syndrome: A Historical Perspective and Call to Action,” in the International Journal of Molecular Sciences. The editorial is authored by members of TREND and other leading experts in Prader-Willi syndrome (PWS) who call for the Food and Drug Administration (FDA) to extend regulatory flexibility in the approval of new drugs for this rare genetic disorder.
Clinical trials for PWS have shown promise with potential treatments, but FDA approval of these therapeutics remains elusive. Even though extensive literature has been published about the life-threatening medical issues and social burdens of PWS, the last treatment approved was for human growth hormone therapy in 2000. As with other rare disease trials, there are inherent challenges in developing sufficient study endpoints and achieving an adequate sample size to reach statistical significance in findings. However, the FDA has exercised its authority to approve drugs for other rare disease populations even when the drugs did not meet their primary clinical endpoints; they have not done this for PWS.
Therapies in recent and current trials have shown great benefit for those with PWS and yet face continued obstacles from the FDA. A recent example cited in the editorial is that of diazoxide choline controlled release (DCCR), which has shown benefits and a favorable safety profile and yet has not been approved. These FDA denials are of great concern to patients, caregivers, and clinicians alike, and the consequences for these individuals and their families are significant. The authors of this editorial ask for equity in considerations for the PWS population to pave the way for treatment approvals that will improve safety and quality of life.
This editorial in the International Journal of Molecular Sciences can be found at https://www.mdpi.com/1422-0067/24/14/11574.
For additional information about this editorial, please contact corresponding author Deepan Singh at desingh@maimonidesmed.org, and for collaboration and partnerships with TREND Community, please contact Maria Picone at interested@trend.community.