Zevra Therapeutics Secures FDA Approval for Miplyffa, a Breakthrough Treatment for Niemann-Pick Disease Type C

FDA Approval:
Zevra Therapeutics' Miplyffa (arimoclomol) has received FDA approval as the first treatment for Niemann-Pick disease type C (NPC), an ultra-rare and progressive neurodegenerative disorder.

Indication:
Miplyffa is indicated for use in combination with miglustat for the treatment of neurological manifestations of NPC in adults and pediatric patients 2 years of age and older.

Rare Pediatric Disease Priority Review Voucher:
Zevra Therapeutics has been awarded a rare pediatric disease priority review voucher in conjunction with the approval.

AmplifyAssist Patient Support Program:
Zevra has launched the AmplifyAssist patient support program to assist eligible patients and their caregivers with various needs, including insurance education, co-pay assistance, and disease management counseling.

Clinical Evidence:
The FDA approval was based on comprehensive data from a New Drug Application (NDA), including a 48-month open-label extension study that suggested improved outcomes compared to a matched National Institutes of Health NPC natural history cohort.

Availability:
Zevra plans to initiate launch activities immediately, with Miplyffa expected to be commercially available in the U.S. within eight to twelve weeks.

Disease Impact:
NPC is an ultra-rare, relentlessly progressive, and fatal neurodegenerative disease affecting approximately 900 people in the U.S., with varying clinical presentations in both children and adults.

Treatment Mechanism:
Miplyffa enhances the activation of specific transcription factors, leading to increased expression of lysosomal regulatory genes and lowering unesterified cholesterol levels in NPC patient cells.