International Niemann–Pick Disease Alliance
Update on the Clinical Trial of VTS-270: The NPC Study
November 2016 Newsletter
Update on the Vtesse Clinical Trial of VTS-270:
Currently, we have enrolled 38 patients into the trial across 17 sites worldwide. In addition to enrolling patients within countries with active clinical trial sties, and we continue to recruit patients from all over the world to come into countries with active trial sites.
Many families are traveling great distances for the opportunity to participate in the trial and some have even relocated to the United States for the year of the trial. To everyone involved in the trial, we sincerely thank you for your contributions to finding answers for NPC patients and their families.
We ask for your support as we strive to reach the goal of enrolling 51 patients in the trial.
VTS-270 Receives a Pediatric Disease Designation from FDA:
On November 29th, Vtesse announced that the U.S. Food and Drug Administration (FDA) granted Rare Pediatric Disease designation to VTS-270. The Rare Pediatric Disease designation applies to drugs designed to treat serious or life- threatening rare diseases afflicting fewer than 200,000 people in the United States in which the serious or life-threatening manifestations primarily affect individuals aged from birth to 18 years. Under this program, a sponsor who receives an approval for a drug or biologic for a rare pediatric disease and meets additional criteria may qualify for a priority review voucher that can be redeemed to receive a priority review of a subsequent marketing application for a different product.
To find out more about the trial and to find a clinical trial site please visit www.theNPCstudy.com or https://clinicaltrials.gov/ct2/show/NCT02534844?term=vtesse&rank=1