Therapy Areas: Central Nervous System
Avrobio Gains FDA Clearance of Investigational New Drug Application for AVR-RD-01 Gene Therapy for the Treatment of Fabry Disease
30 April 2019 - - The US Food and Drug Administration has cleared US-based gene therapy company Avrobio, Inc.'s (NASDAQ: AVRO) Investigational New Drug application for AVR-RD-01, its gene therapy candidate for the treatment of Fabry disease, the company said.

The company now expects to move forward on two key initiatives in its Fabry clinical program: the incorporation of Avrobio's plato platform into its FAB-201 Phase 2 trial, and the dosing of patients at clinical sites in the US.

The plato platform consists of a four-plasmid vector system, automation of a closed cell manufacturing process and a conditioning regimen that utilizes therapeutic drug monitoring.

Clinical data presented at WORLDSymposium in February 2019 continued to support the potential of AVR-RD-01 as a gene therapy for Fabry disease, the company said.

A total of 7 patients have been dosed with AVR-RD-01 across the investigator-sponsored Phase 1 clinical study and FAB-201, the Phase 2 clinical trial of AVR-RD-01 in Fabry disease, which is currently underway in Australia.

The company plans to provide additional preliminary clinical data from these trials this summer.

AVR-RD-01 is an ex vivo lentiviral gene therapy candidate being investigated as a single-dose therapy with the potential to provide durable and potentially life-long therapeutic benefit for patients with Fabry disease.

AVR-RD-01 is designed to employ a lentiviral vector system that is an efficient gene transfer technology for the permanent integration of functional copies of the gene into the patient's own stem cells.

In patients with Fabry disease, hematopoietic stem cells are collected from the patient, and then transduced with lentiviral vector carrying a functional version of the GLA gene that encodes active α-galactosidase A the enzyme that is deficient in Fabry disease to create AVR-RD-01 gene therapy.

AVR-RD-01 is then infused back into the patient with the goal of restoring normal GLA gene expression such that functional AGA enzyme is sufficiently produced by the patient's own body.

FAB-201 is an open-label, multinational study of the efficacy and safety of AVR-RD-01 in approximately 8 to 12 treatment-naïve males, aged 16 years and older, with classic Fabry disease.

The subjects will have a confirmed diagnosis of classic Fabry disease based on deficient AGA enzyme activity who have not yet received treatment with enzyme replacement therapy within the past ten years and/or chaperone therapy at the time of screening.

The study will expand into the US in the second half of 2019 and includes five study periods: Screening, Baseline, Pre-transplant, Transplant, and Post-transplant Follow-up at 48 weeks.

Patients will undergo a conditioning regimen administration with busulfan intravenously for four days to achieve myeloablation prior to the transplant.

After study completion, consenting subjects will continue periodic safety and efficacy assessments for approximately 14 years, for a total of 15 years post-transplant follow-up.

The plato platform, Avrobio's commercial-scale platform for anticipated future worldwide commercialization and pipeline expansion activities, consists of a proprietary four-plasmid vector system, automation of a closed cell manufacturing process and a conditioning regimen that utilizes therapeutic drug monitoring.

The plato platform is designed to enhance the potency, safety, efficacy, and long-term durability of Avrobio's gene therapies, and may additionally provide the capability to address central nervous system manifestations that accompany many lysosomal storage diseases.

The platform is additionally being incorporated in the planned Phase 1/2 clinical trial of AVR-RD-02 (GAU-201) in Canada and Australia. That trial is expected to begin in the second half of 2019.

Avrobio, Inc., is a Phase 2 clinical-stage gene therapy company developing gene therapies to potentially cure rare diseases with a single dose.

The company's lentiviral-based gene therapies employ hematopoietic stem cells that are collected from the patient and then modified with a lentiviral vector to insert a functional copy of the gene that is defective in the target disease.

Avrobio is focused on the development of its gene therapy, AVR-RD-01, in Fabry disease, as well as additional gene therapy programs in other lysosomal storage disorders including Gaucher disease, Cystinosis and Pompe disease. Avrobio is headquartered in Cambridge, MA and has offices in Toronto, ON.
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