The European Commission has granted Orphan Designation for PBKR03, a treatment from Passage Bio to treat Krabbe disease. Krabbe disease is a rare lysosomal storage disorder that currently does not have any approved disease-modifying treatments. The approval comes after the U.S. Food and Drug Administration’s (FDA) Fast Track Designation approval, following positive reviews from the European Medicines Agency (EMA). Bruce Goldsmith, Ph.D., President and CEO of Passage Bio, said, “We are planning to initiate in the first half of this year our clinical trial program, GALax-C, to study PBKR03 as a treatment for early infantile Krabbe disease, the most common and severe form of the disorder. We’re pleased to receive Orphan designation from the European Commission, which — along with multiple FDA designations — reinforces the potential of our approach and the urgent unmet medical need for treatments for infants afflicted with this devastating disease.”
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