New Medicines

Fabry's disease


Advanced therapy medicinal product (ATMP)
Freeline Therapeutics
Freeline Therapeutics

Development and Regulatory status

Phase II Clinical Trials
Phase II Clinical Trials
Mar 20 · Granted orphan drug status in EU [3].


A next generation liver directed adeno associated virus (AAV) based gene therapy.
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].
Fabry's disease
Intravenous infusion

Trial or other data

Feb 20 · PI/II MARVEL1 trial is recruiting [2].
Jul 19 · Freeline Therapeutics initiates the PI/II MARVEL1 trial to assess the safety and efficacy of FLT190, for the treatment of Fabrys disease (NCT04040049; FLT190-01). Besides safety, the study will explore the potential of FLT 190 in treating the signs and symptoms of Fabry´s disease, following a single administration, including αGLA activity levels, Gb3/Lyso-Gb3 clearance, changes in Gb3 in renal and skin biopsies, renal and cardiac function, GLA immune response, viral shedding and quality of life. The baseline-controlled, non-randomised, open-label, single-ascending dose study intends to enrol 15 adults in Italy, Norway, and the UK (at the Royal Free Hospital in London) Collection of primary outcome data is due to complete Dec 21 [2].