AVR RD 01

Unassigned

New Medicines

Males >16 years with a confirmed diagnosis of classic Fabry's disease based on deficient AGA enzyme activity who have not previously received treatment with enzyme replacement therapy (ERT) and/or chaperone therapy within 3 years of the time of screening

Information

New molecular entity
AVROBIO
AVROBIO

Development and Regulatory status

None
None
Phase II Clinical Trials
Yes
Yes
Nov 20ARVOBIO announces intention to submit its briefing book to the US FDA to obtain accelerated approval for AVR RD 01 in Fabry´s disease.[2]
Oct 20EMA grant Orphan Drug status for Fabry´s disease in EU. [2]
Dec 18US FDA granted orphan drug designation.[2]

Category

Gene therapy to be given as a single, one-off dose. Autologous haematopoietic stem cells are collected, genetically modified ex vivo using a lentiviral vector containing the functional GLA gene. Cells are infused back into the patient. [2]
Anderson-Fabry disease is an X-linked recessive inborn error of glycosphingolipid metabolism caused by deficiency of alpha-galactosidase A. It is estimated to occur in 1 in 55,000 males in the classical form but the atypical variant may be more common [1].
Males >16 years with a confirmed diagnosis of classic Fabry's disease based on deficient AGA enzyme activity who have not previously received treatment with enzyme replacement therapy (ERT) and/or chaperone therapy within 3 years of the time of screening
Intravenous infusion

Trial or other data

Feb 21Company announced a 100% reduction/complete clearance (p<0.0001) of toxic substrate in the kidney biopsy (which is the primary outcome measure) of the first patient dosed with the plato® gene therapy platform in the ongoing PII trial of AVR-RD-01. Interim data from 4 pts in the trial has previousely showed that the 1st pt produced an endogenous supply of functional AGA enzyme up to 22 months post-treatment. The 3rd and 4th patients dosed are also showing good signs of efficacy. AVR RD 01 has been generally well tolerated and no safety events related to AVR RD 01 reported. Four serious adverse events (relating to conditioning regimen with melphalan, stem cell mobilization, underlying disease or pre-existing conditions) were reported but were not attributed to AVR RD 01. The trial completes in Nov 2021. [2-4]
Feb 18PII open label, single arm trial in Australia, Canada and the US (NCT03454893) started to evaluate the efficacy and safety of AVR RD 01, in males aged >16 who have not previously received treatment with enzyme replacement therapy (ERT) or other therapies for Fabry disease. The primary efficacy endpoint is change from baseline in the average number of Gb3 inclusions per peritubular capillary (PTC)3 as measured in a kidney biopsy 1 year post-treatment. [2,3]