Regulatory Decision Summary for Galafold

Galafold was studied against enzyme replacement therapy (ERT) in ERT-experienced patients with Fabry disease and against placebo in ERT-naïve patients with Fabry disease. Galafold stabilized renal function (GFR) for up to 30 months. Its effect on renal function was better than placebo-treated patients and comparable to ERT. Long-term treatment with Galafold also led to significant reductions in Left Ventricular Mass Indices, and a lower incidence of clinical composite events (renal, cardiac, cerebrovascular or deaths) compared to the ERT treatment. Treatment with Galafold was associated with reductions of disease substrates (urine GL-3 and plasma lyso-Gb₃).

Galafold was shown to be generally safe and well tolerated. The most common adverse drug reaction, occurring in >10% of patients was headache. Adverse events were generally mild to moderate in severity and not related to treatment. Two patients experienced serious adverse events considered possibly related to Galafold treatment: proteinuria in one subject, and paraesthesia and fatigue in another.

An important uncertainty is whether all patients with amenable mutations would respond adequately. Also, there is a potential for Galafold to worsen the disease condition in patients who do not have amenable mutations, who administer the wrong dosage or more frequent regimens, or who have severe renal impairment.

Safety concerns and uncertainties were addressed in the Canadian Product Monograph and Risk Management Plan.

Based on the totality of the data, Galafold has a positive benefit-risk-uncertainty profile for adults with a confirmed diagnosis of Fabry disease and who have a mutation determined to be amenable by an in vitro assay.

For more information on Health Canadas decision, please view the Summary Basis of Decision.