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Food and Drug Administration accelerated approval for GENGLYCOS (pariglasgene brecaparvovec-opnr) arrived Tuesday for Ultragenyx Pharmaceutical Inc. (Nasdaq Global Select Market: RARE), the company disclosed in an 8-K.
The therapy, also designated DTX401, is cleared for adult and pediatric patients eight years of age and older with glycogen storage disease type Ia.
The FDA decision rests on the Phase 3 GlucoGene study, a 48-week randomized, double-blind, placebo-controlled trial enrolling 46 participants.
Patients received DTX401 at a dose of 1.0 x 10^13 genome copies per kilogram or placebo.
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