PASG - Passage Bio shares rise on European Commission orphan status for PBKR03

Passage Bio (PASG) shares up nearly 12% premarket after the company announces that the European Commission has granted Orphan designation for PBKR03, an adeno-associated virus ((AAV))-delivery gene therapy for the treatment of Krabbe disease (Globoid Cell Leukodystrophy), a rare and often life-threatening lysosomal storage disease.The designation was based on a positive opinion from the European Medicines Agency Committee for Orphan Medicinal Products.Passage Bio expects to initiate a global Phase1/2 clinical trial, GALax-C, for PBKR03 in the first half of this year, with Initial safety and biomarker data expected in late 2021 or early 2022. The trial is designed as a dose-escalation study of a single ICM dose of PBKR03 in pediatric patients with early infantile Krabbe disease.

For further details see:

Passage Bio shares rise on European Commission orphan status for PBKR03