Zevra's MIPLYFFA Shows Positive Data in Infants with Niemann-Pick Disease Type C
ZVRA sits 73% above its 52-week low of $7.16.
Summary
Zevra Therapeutics announced the publication of positive data for its approved drug, MIPLYFFA, from a pediatric substudy in infants with Niemann-Pick Disease Type C (NPC). The study found MIPLYFFA was well tolerated in infants aged 12 to <24 months, with no new safety signals observed. This data supports the potential for earlier treatment initiation in a younger patient population, expanding on its current FDA approval for patients aged 2 years and older. This positive development could increase the addressable market for MIPLYFFA and reinforces its clinical utility for this rare disease.
At the time of this announcement, ZVRA was trading at $12.42 on NASDAQ in the Life Sciences sector, with a market capitalization of approximately $747.8M. The 52-week trading range was $7.16 to $14.27. This news item was assessed with positive market sentiment and an importance score of 7 out of 10. Source: GlobeNewswire.