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

Chronic treatment of Acute Hepatic Porphyria (AHP)

Initial treatment

Patient must have a confirmed diagnosis of AHP based on clinical features; AND

Patient must have a previous result that showed a 4 times greater than the upper limit of normal (ULN) on one of the following tests: (i) urinary porphobilinogen, (ii) plasma porphobilinogen, (iii) delta-aminolevulinic acid; AND

Patient must have had at least 2 porphyria attacks (requiring hospitalisation, presentation at an emergency department, or medical intervention via a porphyria clinic) within the 6 months prior to: (i) commencement of this drug; or (ii) prophylactic treatment with gonadotropin-releasing hormone analogues or hemin; AND

Patient must not have previously undergone a liver transplant; AND

The treatment must be initiated under the supervision of a healthcare professional experienced in the management of porphyria; AND

Patient must be at least 12 years of age.

For the purposes of administering this restriction, clinical features to confirm a diagnosis of AHP can include but are not limited to acute attacks of: abdominal, back, chest, extremities, and/or limb pain.

At the time of the authority application, prescribers should request the appropriate number of vials for a single dose 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 and must include:

(1) The baseline number of porphyria attacks within a defined 6-month period [dates of the 6-month period must be included].

(2) Pathology measurements that indicate 4 times greater than the ULN of one of the following tests:

(i) urinary porphobilinogen;

(ii) plasma porphobilinogen;

(iii) delta-aminolevulinic acid.

Date of pathology report and unique identifying number/code that links the pathology result to the individual patient must be included.

(3) Current weight of the patient.

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

Chronic treatment of Acute Hepatic Porphyria (AHP)

Continuing treatment

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

Patient must demonstrate improvement in the frequency or severity of AHP attacks from baseline while on treatment with this drug; AND

Must be treated by a healthcare professional experienced in the management of porphyria or in consultation with a healthcare professional experienced in the management of porphyria; AND

Patient must not have previously undergone a liver transplant; AND

Patient must be at least 12 years of age.

The number of AHP attacks while on treatment must be documented in the patient's medical records.

At the time of the authority application, prescribers should request the appropriate number of vials for a single dose 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).