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

Paroxysmal nocturnal haemoglobinuria (PNH)

Initial treatment - Initial 1 (new patient) induction dose

Patient must not have received prior treatment with this drug for this condition; AND

Patient must have a diagnosis of PNH established by flow cytometry; AND

Patient must have a PNH granulocyte clone size equal to or greater than 10%; AND

Patient must have a raised lactate dehydrogenase value at least 1.5 times the upper limit of normal; AND

Patient must have experienced a thrombotic/embolic event which required anticoagulant therapy; or

Patient must have been transfused with at least 4 units of red blood cells in the last 12 months; or

Patient must have chronic/recurrent anaemia, where causes other than haemolysis have been excluded, together with multiple haemoglobin measurements not exceeding 70 g/L in the absence of anaemia symptoms; or

Patient must have chronic/recurrent anaemia, where causes other than haemolysis have been excluded, together with multiple haemoglobin measurements not exceeding 100 g/L in addition to having anaemia symptoms; or

Patient must have debilitating shortness of breath/chest pain resulting in limitation of normal activity (New York Heart Association Class III) and/or established diagnosis of pulmonary arterial hypertension, where causes other than PNH have been excluded; or

Patient must have a history of renal insufficiency, demonstrated by an eGFR less than or equal to 60 mL/min/1.73m2, where causes other than PNH have been excluded; or

Patient must have recurrent episodes of severe pain requiring hospitalisation and/or narcotic analgesia, where causes other than PNH have been excluded; AND

The treatment must be the sole PBS-subsidised therapy for this condition; AND

Must be treated by a haematologist; or

Must be treated by a non-specialist medical physician who has consulted a haematologist on the patient's drug treatment details.

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) details of the proposed prescription(s); 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).

At the time of the authority application, medical practitioners should request the appropriate number of vials for a single loading dose based on the patient's weight, as per the Product Information

At the time of the authority application, details (result and date of result) of the following monitoring requirements must be provided:

(i) Haemoglobin (g/L)

(ii) Platelets (x10⁹/L)

(iii) White Cell Count (x10⁹/L)

(iv) Reticulocytes (x10⁹/L)

(v) Neutrophils (x10⁹/L)

(vi) Granulocyte clone size (%)

(vii) Lactate Dehydrogenase (LDH)

(viii) the upper limit of normal (ULN) for LDH as quoted by the reporting laboratory

(ix) the LDH:ULN ratio (in figures, rounded to one decimal place) must be at least 1.5

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

Paroxysmal nocturnal haemoglobinuria (PNH)

Initial treatment - Initial 2 (switch from LSDP eculizumab) induction dose

Patient must have previously received eculizumab for the treatment of this condition funded under the Australian Government's Life Saving Drugs Program (LSDP); AND

Patient must have a diagnosis of PNH established by flow cytometry prior to LSDP-funded treatment with eculizumab; AND

Patient must have a PNH granulocyte clone size equal to or greater than 10% prior to LSDP-funded treatment with eculizumab; AND

Patient must have a raised lactate dehydrogenase value at least 1.5 times the upper limit of normal prior to LSDP-funded treatment with eculizumab; AND

Patient must have experienced a thrombotic/embolic event which required anticoagulant therapy prior to LSDP-funded treatment with eculizumab; or

Patient must have been transfused with at least 4 units of red blood cells in the last 12 months prior to LSDP-funded treatment with eculizumab; or

Patient must have chronic/recurrent anaemia, where causes other than haemolysis have been excluded, together with multiple haemoglobin measurements not exceeding 70 g/L in the absence of anaemia symptoms prior to LSDP-funded treatment with eculizumab; or

Patient must have chronic/recurrent anaemia, where causes other than haemolysis have been excluded, together with multiple haemoglobin measurements not exceeding 100 g/L in addition to having anaemia symptoms prior to LSDP-funded treatment with eculizumab; or

Patient must have debilitating shortness of breath/chest pain resulting in limitation of normal activity (New York Heart Association Class III) and/or established diagnosis of pulmonary arterial hypertension, where causes other than PNH have been excluded prior to LSDP-funded treatment with eculizumab; or

Patient must have a history of renal insufficiency, demonstrated by an eGFR less than or equal to 60 mL/min/1.73m2, where causes other than PNH have been excluded prior to LSDP-funded treatment with eculizumab; or

Patient must have recurrent episodes of severe pain requiring hospitalisation and/or narcotic analgesia, where causes other than PNH have been excluded prior to LSDP-funded treatment with eculizumab; AND

The treatment must be the sole PBS-subsidised therapy for this condition; AND

Must be treated by a haematologist; or

Must be treated by a non-specialist medical physician who has consulted a haematologist on the patient's drug treatment details.

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) details of the proposed prescription(s); 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).

At the time of the authority application, medical practitioners should request the appropriate number of vials for a single loading dose based on the patient's weight, as per the Product Information

At the time of the authority application, details (result and date of result) of the following monitoring requirements must be provided:

(i) Haemoglobin (g/L)

(ii) Platelets (x10⁹/L)

(iii) White Cell Count (x10⁹/L)

(iv) Reticulocytes (x10⁹/L)

(v) Neutrophils (x10⁹/L)

(vi) Granulocyte clone size (%)

(vii) Lactate Dehydrogenase (LDH)

(viii) the upper limit of normal (ULN) for LDH as quoted by the reporting laboratory

(ix) the LDH:ULN ratio (in figures, rounded to one decimal place) must be at least 1.5

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

Paroxysmal nocturnal haemoglobinuria (PNH)

Return from PBS-subsidised eculizumab - induction dose

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

Patient must have received prior PBS-subsidised treatment with eculizumab through the 'Initial treatment - Initial 2 (switching from PBS-subsidised ravulizumab for pregnancy)' criteria; AND

The treatment must be the sole PBS-subsidised therapy for this condition; AND

Must be treated by a haematologist; or

Must be treated by a non-specialist medical physician who has consulted a haematologist on the patient's drug treatment details.

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) details of the proposed prescription(s); 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).

At the time of the authority application, medical practitioners should request the appropriate number of vials for a single loading dose based on the patient's weight, as per the Product Information

Patient may qualify under this treatment phase more than once for the purposes of family planning. Where long-term continuing PBS-subsidised treatment with this drug is planned, a 'Returning' patient may proceed under the 'Subsequent Continuing Treatment' criteria.

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

Paroxysmal nocturnal haemoglobinuria (PNH)

Switching from PBS-subsidised pegcetacoplan or iptacopan - induction doses

Patient must have received PBS-subsidised treatment with either: (i) pegcetacoplan, (ii) iptacopan for this condition; AND

Patient must have developed resistance or intolerance to either: (i) pegcetacoplan, (ii) iptacopan; AND

The treatment must be the sole PBS-subsidised therapy for this condition; AND

Must be treated by a haematologist; or

Must be treated by a non-specialist medical physician who has consulted a haematologist on the patient's drug treatment details.

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) details of the proposed prescription(s); 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).

For continuing PBS-subsidised treatment with this drug, a 'Returning' patient must proceed under the'Subsequent Continuing Treatment' criteria.