Zevra Therapeutics (NASDAQ:ZVRA), has announced FDA approval for its Miplyffa oral capsules, used in combination with miglustat, for the treatment of Niemann-Pick disease C (NPC) in adult and pediatric patients aged two and older. Additionally, Miplyffa was granted a Rare Pediatric Disease Priority Review Voucher.
The FDA approval was based on comprehensive data from the NDA, which included evidence supporting trial endpoints, FDA-preferred analyses, and additional confirmatory clinical and nonclinical evidence. Zevra aims to initiate launch activities for Miplyffa in the U.S. by late 2024.
In a statement, Neil McFarlane, president and CEO of Zevra, said, “NPC is an ultra-rare, relentlessly progressive, degenerative and fatal disease for which there were no FDA-approved treatment options until today. The approval of Miplyffa is a monumental milestone for NPC patients and their family members in the US. We are immensely grateful for the unwavering support we have received over the years from the families and individuals impacted by NPC as well as the collaborative efforts of advocacy groups, researchers and clinicians.”
