Jul. 14 at 1:36 PM
$PHAR As Pharming advances patient recruitment in the FALCON study, napazimone (KL1333) reflects the strategic value of the Abliva acquisition and its role in expanding PHAR’s rare disease pipeline into mitochondrial DNA (mtDNA)-driven primary mitochondrial disease.
The investigational late-stage program combines a novel mechanism of action, an ongoing pivotal study, FDA-agreed primary endpoints, and a positive interim analysis—features that provide important clinical and regulatory visibility at this stage.
With more than 30,000 potentially addressable diagnosed mtDNA mitochondrial disease patients across the U.S., EU4, and the U.K., napizimone (KL1333) is being developed for a rare disease area with significant unmet medical need.
Pharming has clinical development, regulatory, market access, and patient services infrastructure to support continued development and potential future commercialization of napazimone (KL1333) while leveraging its existing rare disease capabilities.
