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

Primary hyperoxaluria type 1

Continuing treatment

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

Patient must continue to demonstrate clinical benefit as assessed by the treating physician; AND

Patient must not have previously undergone liver transplant for primary hyperoxaluria type 1; AND

Must be treated by a nephrologist with experience in the management of hyperoxaluria; or

Must be treated by a paediatrician with experience in the management of hyperoxaluria; or

Must be treated by an authorised prescriber in consultation with one of the above specialty types.

At the time of the authority application, prescribers should request the appropriate number of vials based on the patient's weight, as per the TGA approved Product Information. Up to 1 repeat may be requested for continuing treatment.

The authority application must be made via the Online PBS Authorities System (real time assessment), or in writing via HPOS form upload or mail.

If the application is submitted through HPOS form upload or mail, it must include:

(i) details of the proposed prescription; and

(ii) 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

Primary hyperoxaluria type 1

Transitioning from non-PBS to PBS-subsidised supply - Grandfather arrangements

Patient must have received non-PBS-subsidised treatment with this drug for this condition prior to 1 October 2025; AND

The condition must be primary hyperoxaluria type 1 confirmed by genetic testing; AND

Patient must have undergone treatment with pyridoxine therapy prior to commencing non-PBS-subsidised treatment with this drug for this condition; or

Patient must have a PH1 allele that is considered not responsive to pyridoxine therapy; or

Patient must have been contraindicated for pyridoxine therapy as determined by the treating or consulting clinician prior to commencing non-PBS-subsidised treatment with this drug for this condition; AND

Patient must have had urinary oxalate of at least 0.70 mmol/24 h/1.73 m2 measured by mean 24-h urinary oxalate excretion from a valid 24-h urine collection prior to commencing treatment with this drug for this condition; or

Patient must have urinary oxalate:creatinine ratio greater than the upper limit of normal based on age on at least two of three single-void collections during screening prior to commencing treatment with this drug for this condition; or

Patient must have, prior to commencing treatment with this drug for this condition, clinical symptoms indicative of hyperoxaluria, such as (i) nephrocalcinosis; (ii) renal stones; (iii) renal impairment; (iv) systemic oxalosis; AND

Patient must continue to demonstrate clinical benefit as assessed by the treating physician; AND

Patient must not have previously undergone liver transplant for primary hyperoxaluria type 1; AND

Must be treated by a nephrologist with experience in the management of hyperoxaluria; or

Must be treated by a paediatrician with experience in the management of hyperoxaluria; or

Must be treated by an authorised prescriber in consultation with one of the above specialty types.

At the time of the authority application, prescribers should request the appropriate number of vials based on the patient's weight, as per the TGA-approved Product Information.

The authority application must be made via the Online PBS Authorities System, or in writing via HPOS form upload or mail.

If the application is submitted through HPOS form upload or mail, it must include:

(i) details of the proposed prescription; and

(ii) 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).