Listing of Pharmaceutical Benefits (NHL) - Schedule 4 part 1

Fabry disease

Initial treatment

Patient must have at least one of: (i) documented deficiency of alpha-galactosidase enzyme activity in blood, (ii) presence of genetic mutations known to result in deficiency of alpha-galactosidase enzyme activity; AND

Patient must have a documented migalastat amenable galactosidase alpha (GLA) gene variant; AND

Patient must have an estimated glomerular filtration rate (eGFR) of at least 30 mL/min/1.73 m2; AND

Patient must be male with Fabry-related renal disease confirmed by at least one of the following: (i) abnormal albuminuria of more than 20 mcg/min, as determined by 2 separate samples at least 24 hours apart, (ii) abnormal proteinuria of more than 150 mg/24 hours, (iii) albumin:creatinine ratio greater than upper limit of normal in 2 separate samples at least 24 hours apart, (iv) renal disease due to long-term accumulation of glycosphingolipids in the kidneys; or

Patient must be female with Fabry-related renal disease confirmed by at least one of the following: (i) proteinuria of more than 300 mg/24 hours with clinical evidence of progression, (ii) renal disease due to long-term accumulation of glycosphingolipids in the kidneys; or

Patient must have Fabry-related cardiac disease confirmed by at least one of the following: (i) left ventricular hypertrophy, as evidenced by cardiac magnetic resonance imaging (MRI) or echocardiogram data, in the absence of hypertension, (ii) significant life-threatening arrhythmia or conduction defect, (iii) late gadolinium enhancement or a low T1 on cardiac MRI; or

Patient must have Fabry-related either: (i) ischaemic disease, (ii) cerebrovascular disease as shown on objective testing with no other cause or risk factors identified; or

Patient must have Fabry-related uncontrolled chronic pain despite the use of recommended doses of appropriate analgesia and antiseizure medications for peripheral neuropathy; or

Patient must have significant Fabry-related gastrointestinal symptoms despite the use of the recommended doses of appropriate pharmacological therapies; AND

Must be treated by a physician with expertise in the management of Fabry disease; AND

Patient must be at least 12 years of age.

If hypertension is present in patients relying their eligibility on Fabry-related cardiac disease, the prescriber must treat it optimally for at least 6 months prior to submitting the first PBS authority application.

Confirmation of eligibility for treatment with diagnostic reports including the confirmed mutations must be documented in the patient's medical records.

The authority application must be made in writing and must include:

(1) details of the proposed prescription; and

(2) a completed authority application form relevant to the indication and treatment phase (the latest version is located on the website specified in the Administrative Advice).

Listing of Pharmaceutical Benefits (NHL) - Schedule 4 part 1

Fabry disease

Continuing treatment

Patient must have received prior PBS-subsidised treatment with this drug for this condition; AND

Patient must have demonstrated clinical improvement or stabilisation of condition, the details of which must be kept with the patient's record; AND

Patient must not have developed another life threatening/severe disease where long term prognosis is unlikely to be influenced by migalastat; AND

Must be treated by a physician with expertise in the management of Fabry disease.

Listing of Pharmaceutical Benefits (NHL) - Schedule 4 part 1

Fabry disease

Grandfather arrangement (transition from LSDP-funded Fabry disease therapy)

Patient must have previously received treatment with this drug for this condition funded under the Australian Government's Life Saving Drugs Program (LSDP) prior to 1 September 2024; or

Patient must have previously received treatment with Enzyme Replacement Therapy for this condition funded under the Australian Government's Life Saving Drugs Program (LSDP) prior to 1 September 2024; AND

Patient must have a documented migalastat amenable galactosidase alpha (GLA) gene variant prior to commencing treatment with this drug; AND

Patient must have/have had an estimated glomerular filtration rate (eGFR) of at least 30 mL/min/1.73 m2 prior to commencing treatment with this drug; AND

Must be treated by a physician with expertise in the management of Fabry disease; AND

Patient must be at least 12 years of age.

A patient may qualify for PBS-subsidised treatment under this restriction once only. For continuing PBS-subsidised treatment, a Grandfathered patient must qualify under the Continuing treatment criteria.

Confirmation of eligibility for treatment with diagnostic reports including the confirmed mutations must be documented in the patient's medical records.

The authority application must be made in writing and must include:

(1) details of the proposed prescription; and

(2) a completed authority application form relevant to the indication and treatment phase (the latest version is located on the website specified in the Administrative Advice).