Apr 21 · Granted orphan drug status in EU [3].

Mar 21 · FDA grants fast track status [3].

Oct 20 · FDA grants orphan drug status and rare paediatric disease (RPD) designation to PBKR 03 for the treatment of globoid cell leukodystrophy (Krabbe ´s disease) [3].

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].

Feb 22 · PI/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].