dm+d

Unassigned

New Medicines

Krabbe's disease

Information

New molecular entity
Passage Bio
Passage Bio

Development and Regulatory status

Phase II Clinical Trials
Phase II Clinical Trials
Phase II Clinical Trials
Apr 21Granted orphan drug status in EU [3].
Mar 21FDA grants fast track status [3].
Oct 20FDA grants orphan drug status and rare paediatric disease (RPD) designation to PBKR 03 for the treatment of globoid cell leukodystrophy (Krabbe ´s disease) [3].

Category

An adeno-associated virus (AAV) based gene therapy using a next-generation proprietary AAVhu68 capsid to deliver DNA encoding galactosylceramidase enzyme to a patients cells
This is an autosomal recessive leukodystrophy. There is a lack of the enzyme galatosylceramidase (GALC) leading to abnormalities of myelin formation in both the central and peripheral nervous system. It is often fatal.Worldwide it is estimated to affect 1 in 100,000-200,000 births, although a higher incidence occurs in certain groups, eg some Israeli communities. Males and females are equally affected [1].
Krabbe's disease
Intracisternal
Parenteral

Trial or other data

Feb 22PI/II GALax-C trial to evaluate the safety, tolerability and efficacy of PBKR03 gene therapy for globoid cell leukodystrophy (Krabbe disease) starts (NCT04771416; PBKR03-001). This open-label, multicenter, dose escalation trial is designed to recruit 24 infants, aged one month-9 months in the US, Brazil, Netherlands, Canada, Israel and the UK (in Manchester). The dose-ranging portion of the study will enroll independent dose escalation cohorts in two age groups of subjects with early infantile Krabbe disease: Cohort 1: 3 subjects aged ≥4 to <9 months will receive the low dose (Dose I); Cohort 2: 3 subjects aged ≥4 to <9 months will receive the high dose (Dose II); Cohort 3: 3 subjects aged ≥1 to <4 months will receive the low dose (Dose I); Cohort 4: 3 subjects aged ≥1 to 1 to <9 months. These subjects will receive a dose chosen based on the data obtained in part 1 of the study This will be a 2-year study with a 3-year safety extension. Primary outcomes include change from baseline in nerve conduction and velocity in motor and sensory nerve conduction studies and adverse events; collection of these data is due to complete Jan 27 [2].