The U.S. Food and Drug Administration approved the first treatment for Niemann-Pick disease type C.
“It has been almost 30 years to the day since Michael, Marcia and Christa Parseghian were diagnosed with Niemann-Pick Type C disease, eventually taking their young lives. Since that time the Parseghian family and so many other families, researchers and volunteers have worked tirelessly to advance our understanding of NPC and bring treatments to those affected by this devastating disease. Today marks a momentous occasion with the FDA approval of Miplyffa and the start of a new era with the first official medication for the treatment of NPC disease. We will continue to fight for NPC families until this disease is eradicated,” Director of The Ara Parseghian Medical Research Fund, Sean Kassen said.
NPC is a rare genetic disease that results in progressive neurological symptoms and organ dysfunction. It is caused by changes in either the NPC1 or NPC2 gene, affecting the necessary transport of cholesterol and other lipids within a cell. As a result, these cells do not function as they should, ultimately causing organ damage. On average, individuals affected by this devastating disease only live for about 13 years.
A heartfelt message was delivered to families, supporters, researchers on Friday morning after receiving the news. “This milestone is not just a victory for those impacted by NPC disease; it is a beacon of hope for all the families who have faced this relentless disease with courage and resilience. For years, we have watched too many struggle with the devastating effects of NPC, holding onto hope for a breakthrough that seemed so elusive. Today, that hope is no longer a distant dream but a tangible reality.”
Expressing gratitude and continued movement in the search for a cure, Cindy Parseghian and Sean Kassen shared the following:
“To the researchers, scientists, and medical professionals who have dedicated their lives to finding a cure, thank you for your relentless pursuit of knowledge and your commitment to making the impossible possible. To the families and patients who have bravely participated in trials and shared their experiences, your contributions have been invaluable and your bravery has paved the way for this monumental achievement. To our generous benefactors, your gifts have led to this transformative moment. Today, we celebrate this victory with tears of joy and hearts full of gratitude. But our work is not done. We must continue to support ongoing research, advocate for access to treatments, and provide unwavering support to those affected by NPC. Together, we will continue to push the boundaries of what is possible and push towards a cure.”
Read the full FDA release along with the statement from Zevra Therapeutics.
Learn more about Miplyffa at https://miplyffa.com/