FDA Greenlights Zevra Therapeutics’ MIPLYFFA for Niemann-Pick Disease Type C
Zevra Therapeutics, Inc. has announced that the FDA has approved MIPLYFFA (arimoclomol) capsules as the first oral treatment for Niemann-Pick disease type C. Indicated for use alongside miglustat, MIPLYFFA is intended for both adult and pediatric patients aged two and older. This approval marks a significant milestone for NPC patients, who previously had no FDA-approved options. Zevra has also received a rare pediatric disease priority review voucher in conjunction with this approval.
Neil F. McFarlane, President and CEO of Zevra Therapeutics, emphasized the importance of this approval...
