dm+d

Unassigned

New Medicines

Fabry's disease

Information

New molecular entity
Freeline Therapeutics
Freeline Therapeutics

Development and Regulatory status

Phase II Clinical Trials
Phase II Clinical Trials
None
Yes
Yes
May 20Granted orphan drug status in US [4].
Mar 20Granted orphan drug status in EU [3].

Category

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 20Preliminary data from the PI/II MARVEL1 trial on the starting dose in a dose escalation study show that the infusion was well tolerated. A 3 to 4-fold increase in plasma αGLA activity was achieved by week 4 (0.3 → 1 ± 0.2 nmol/hr/ml) and sustained through the data cutoff at week 20 [5].
Feb 20PI/II MARVEL1 trial is recruiting [2].
Jul 19Freeline 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].