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

Primary hyperoxaluria type 1

Initial treatment - loading dose

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

Patient must have undergone treatment with pyridoxine therapy; or

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

Patient must be contraindicated for pyridoxine therapy as determined by the treating or consulting clinician; AND

Patient must have urinary oxalate greater than or equal to 0.70 mmol/24 h/1.73 m2 measured by mean 24-h urinary oxalate excretion from a valid 24-h urine collection; 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; or

Patient must have clinical symptoms indicative of hyperoxaluria, such as (i) nephrocalcinosis; (ii) renal stones; (iii) renal impairment; (iv) systemic oxalosis; 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 2 repeats may be requested for initial treatment.

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).