Table 2 Expert-based recommendations on the use of migalastat in Fabry disease (FD)

According to current evidence, migalastat is an effective and generally well tolerated treatment for FD in patients with amenable pathogenic mutations.

The use of oral therapy with migalastat can improve the quality of life of patients with FD.

In a male patient aged ≥16 years with amenable mutations and type 1 classic FD, migalastat may also be considered at diagnosis when signs/symptoms of organ damage are not present.

In a male patient aged ≥16 years with amenable mutations and type 2 late-onset FD, migalastat may also be considered at diagnosis at the presence of signs and symptoms of organ damage.

In a female patient aged ≥16 years, with amenable mutations and type 1 classic or type 2 late-onset FD, migalastat can be considered at the presence of early signs/symptoms of organ involvement.

Treatment with migalastat can be considered in patients with FD aged ≥16 years with amenable mutations, and heart hypertrophy and/or rhythm alterations and/or ECG alterations.

Treatment with migalastat can be considered in patients with FD aged ≥16 years with amenable mutations and persistent microalbuminuria, and/or proteinuria and/or eGFR 30–90 ml/min/1.73m²

Treatment with migalastat can be considered in patients with FD aged ≥16 years with amenable mutations and transient ischemic attack /stroke and/or white matter lesions.

Treatment with migalastat can be considered in patients with FD aged ≥16 years with amenable mutations with acroparaesthesia, and/or gastrointestinal symptoms, and/or hearing loss.

In a patient aged ≥16 years with amenable mutation already in treatment with ERT, switching to migalastat should be considered in the case of unstable patients and/or uncontrolled infusion reactions and/or poor compliance to intravenous therapy.