Commencement information

Column 1

Column 2

Column 3

Provisions

Commencement

Date/Details

1. The whole of this instrument

1 August 2026

1 August 2026

Cipla Ambrisentan

C11229 C13497 C13575 C18737 C18741 C18743 C18752 C18836

See Schedule 2

See Schedule 2

Tablet, dispersible, 125 mg

Oral

Deferasirox Juno

C7374 C7375 C7385 C8326 C8328 C8329 C9222 C9258 C9302

P7385 P8326 P8328 P8329 P9222 P9258 P9302

168

2

C7374 C7375 C7385 C8326 C8328 C8329 C9222 C9258 C9302

P7374 P7375

168

5

Tablet, dispersible, 250 mg

Oral

Deferasirox Juno

C7374 C7375 C7385 C8326 C8328 C8329 C9222 C9258 C9302

P7385 P8326 P8328 P8329 P9222 P9258 P9302

168

2

C7374 C7375 C7385 C8326 C8328 C8329 C9222 C9258 C9302

P7374 P7375

168

5

Nitisinone

Capsule 2 mg

Oral

Orfadin

C18958 C19053 C19057

See Schedule 2

See Schedule 2

Capsule 5 mg

Oral

Orfadin

C18958 C19053 C19057

See Schedule 2

See Schedule 2

Capsule 10 mg

Oral

Orfadin

C18958 C19053 C19057

See Schedule 2

See Schedule 2

Capsule 20 mg

Oral

Orfadin

C18958 C19053 C19057

See Schedule 2

See Schedule 2

Oral suspension 4 mg per mL, 90 mL

Oral

Orfadin

C18959 C19055 C19058

See Schedule 2

See Schedule 2

Pegunigalsidase alfa

Solution for I.V. injection 20 mg in 10 mL

Injection

ELFABRIO

C18929 C18930 C19015 C19045

See Schedule 2

See Schedule 2

C18992 C18993 C18994 C18995

1 dose of 10 mg per kg of patient weight

3

C19011 C19042

1 dose of 10 mg per kg of patient weight

5

Nitisinone

C18958 C18959 C19053 C19055 C19057 C19058

Sufficient for treatment for 4 weeks

5

Pegunigalsidase alfa

C18929 C18930 C19015 C19045

Sufficient for treatment for 4 weeks

5

Burosumab

C13377

X-linked hypophosphataemia

Initial treatment - New patient

Patient must have a documented confirmation of PHEX pathogenic variant; OR

Patient must have a confirmed diagnosis of X-linked hypophosphataemia demonstrated by the presence of all of the following: (i) a serum phosphate concentration below the age adjusted lower limit of normal; (ii) current or historical (for those with growth plate fusion) radiographic X-ray evidence of rickets; (iii) elevated (or inappropriately normal) serum or plasma FGF-23 levels of above the mean of the assay-specific reference range; (iv) renal phosphate wasting demonstrated by a ratio of tubular maximum reabsorption rate of phosphate to glomerular filtration rate (TmP/GFR) according to age specific normal ranges using the second morning urine void and paired serum sample measuring phosphate and creatinine.

Must be treated by a medical practitioner identifying as at least one of the following specialists: (i) paediatric endocrinologist, (ii) paediatric nephrologist, (iii) endocrinologist, (iv) nephrologist.

At the time of authority application, medical practitioners must request the appropriate number of vials of appropriate strength(s) to provide sufficient drug, based on the weight of the patient, adequate for 4 weeks, according to the specified dosage in the approved Product Information (PI). A separate authority prescription form must be completed for each strength requested. Up to a maximum of 5 repeats will be authorised.

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

Compliance with Authority Required procedures

C18950

Tumour-induced osteomalacia

Continuing treatment

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

Patient must have achieved normalisation in serum phosphate levels; OR

The condition must have been reviewed by a second specialist physician with expertise in the management of this condition, confirming that continuing therapy is clinically required.

Must be treated by a medical practitioner identifying as at least one of the following specialists: (i) paediatric endocrinologist, (ii) paediatric nephrologist, (iii) endocrinologist, (iv) nephrologist.

At the time of authority application, medical practitioners must request the appropriate number of vials of appropriate strength(s) to provide sufficient drug, based on the weight of the patient, adequate for 4 weeks, according to the specified dosage in the approved Product Information (PI). A separate authority prescription form must be completed for each strength requested. Up to a maximum of 5 repeats will be authorised.

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

Compliance with Authority Required procedures

C18952

Tumour-induced osteomalacia

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

Patient must have previously received non-PBS-subsidised treatment with this drug for this condition prior to 1 August 2026; AND

Patient must have, prior to initiating treatment with this drug, a confirmed diagnosis of hypophosphataemia in tumour-induced osteomalacia demonstrated by the presence of all of the following: (i) chronic clinical symptoms of fatigue, bone pain, muscle weakness and/or (pseudo) fractures; (ii) elevated (or inappropriately normal) serum or plasma FGF-23 levels above the mean of the assay-specific reference range; (iii) a serum phosphate concentration below the age adjusted lower limit of normal; (iv) renal phosphate wasting demonstrated by a ratio of tubular maximum reabsorption rate of phosphate to glomerular filtration rate (TmP/GFR) according to age specific normal ranges using the second morning urine void and paired serum sample measuring phosphate and creatinine; AND

Patient must not have been a candidate for curative surgical resection, prior to initiating treatment with this drug, as a result of either: (i) inability to locate the causative tumour using all available functional imaging technologies; (ii) inability to completely resect the causative tumour due to size, anatomical location, or other patient contraindications or comorbidities; OR

Patient must have had demonstrated persistent hypophosphataemia despite complete surgical resection of the suspected causative tumour prior to initiating treatment with this drug; AND

Patient must have achieved normalisation in serum phosphate levels; OR

The condition must have been reviewed by a second specialist physician with expertise in the management of this condition, confirming that continuing therapy is clinically required.

Must be treated by a medical practitioner identifying as at least one of the following specialists: (i) paediatric endocrinologist, (ii) paediatric nephrologist, (iii) endocrinologist, (iv) nephrologist.

At the time of authority application, medical practitioners must request the appropriate number of vials of appropriate strength(s) to provide sufficient drug, based on the weight of the patient, adequate for 4 weeks, according to the specified dosage in the approved Product Information (PI). A separate authority prescription form must be completed for each strength requested. Up to a maximum of 5 repeats will be authorised.

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

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.

Compliance with Authority Required procedures

C18953

X-linked hypophosphataemia

Continuing treatment

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

Patient must have achieved normalisation in serum phosphate levels; OR

The condition must have been reviewed by a second specialist physician with expertise in the management of this condition, confirming that continuing therapy is clinically required; AND

Patient must have radiographical evidence of stabilisation/improvement in rickets in patients without growth plate fusion.

Must be treated by a medical practitioner identifying as at least one of the following specialists: (i) paediatric endocrinologist, (ii) paediatric nephrologist, (iii) endocrinologist, (iv) nephrologist.

At the time of authority application, medical practitioners must request the appropriate number of vials of appropriate strength(s) to provide sufficient drug, based on the weight of the patient, adequate for 4 weeks, according to the specified dosage in the approved Product Information (PI). A separate authority prescription form must be completed for each strength requested. Up to a maximum of 5 repeats will be authorised.

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

Compliance with Authority Required procedures

C18982

Tumour-induced osteomalacia

Initial treatment - New patient

Patient must have a confirmed diagnosis of hypophosphataemia in tumour-induced osteomalacia demonstrated by the presence of all of the following: (i) chronic clinical symptoms of fatigue, bone pain, muscle weakness and/or (pseudo) fractures; (ii) elevated (or inappropriately normal) serum or plasma FGF-23 levels above the mean of the assay-specific reference range; (iii) a serum phosphate concentration below the age adjusted lower limit of normal; (iv) renal phosphate wasting demonstrated by a ratio of tubular maximum reabsorption rate of phosphate to glomerular filtration rate (TmP/GFR) according to age specific normal ranges using the second morning urine void and paired serum sample measuring phosphate and creatinine; AND

Patient must not be a candidate for curative surgical resection as a result of either: (i) inability to locate the causative tumour using all available functional imaging technologies; (ii) inability to completely resect the causative tumour due to size, anatomical location, or other patient contraindications or comorbidities; OR

Patient must have demonstrated persistent hypophosphataemia despite complete surgical resection of the suspected causative tumour.

Must be treated by a medical practitioner identifying as at least one of the following specialists: (i) paediatric endocrinologist, (ii) paediatric nephrologist, (iii) endocrinologist, (iv) nephrologist.

At the time of authority application, medical practitioners must request the appropriate number of vials of appropriate strength(s) to provide sufficient drug, based on the weight of the patient, adequate for 4 weeks, according to the specified dosage in the approved Product Information (PI). A separate authority prescription form must be completed for each strength requested. Up to a maximum of 5 repeats will be authorised.

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

Prescribers should, where possible, rule out all other non-hereditary forms of FGF-23 related hypophosphataemia such as Fanconi syndrome, iron-infusion associated hypophosphataemia or post renal transplantation hypophosphataemia, prior to initiating patients on burosumab treatment for this condition.

Compliance with Authority Required procedures

C18992

Moderate to severe Crohn disease

Initial 3 (recommencement of treatment after a break in targeted therapy of more than 12 months)

Patient must have received prior PBS-subsidised treatment with a targeted therapy for this condition; AND

Patient must have had a break in treatment of 12 months or more from the most recently approved PBS-subsidised targeted therapy for this condition; AND

Patient must have confirmed diagnosis of Crohn disease, defined by standard clinical, endoscopic and/or imaging features including histological evidence; AND

Patient must have a Paediatric Crohn Disease Activity Index (PCDAI) Score greater than or equal to 30 while off treatment with targeted therapy; OR

Patient must have a documented history and radiological evidence of intestinal inflammation if the patient has extensive small intestinal disease while off treatment with targeted therapy; AND

Patient must not receive more than 12-14 weeks of treatment under this restriction per authority application.

Must be treated by a gastroenterologist; OR

Must be treated by a consultant physician [internal medicine specialising in gastroenterology]; OR

Must be treated by a consultant physician [general medicine specialising in gastroenterology]; OR

Must be treated by a paediatrician; OR

Must be treated by a specialist paediatric gastroenterologist.

Patient must be aged 6 to 17 years inclusive.

The following information must be provided by the prescriber at the time of application and documented in the patient's medical records:

(a) current PCDAI score and the date of assessment; or

(b) details (date, unique identifying number/code or provider number) of the pathology or diagnostic imaging test(s) used to assess response to therapy for patients with extensive small intestine disease.

For patients assessed as having extensive intestinal inflammation of the small intestines, such evidence of intestinal inflammation includes:

(i) blood: higher than normal platelet count, or, an elevated erythrocyte sedimentation rate (ESR) greater than 25 mm per hour, or, a C-reactive protein (CRP) level greater than 15 mg per L; or

(ii) faeces: higher than normal lactoferrin or calprotectin level; or

(iii) diagnostic imaging: demonstration of increased uptake of intravenous contrast with thickening of the bowel wall or mesenteric lymphadenopathy or fat streaking in the mesentery.

An increase in the maximum quantity to allow for dosing of up to 10 mg per kg body weight with 3 repeats will be authorised.

If fewer than 3 repeats are requested at the time of the application, authority approvals for sufficient repeats to complete the 3 doses of this drug may be requested through the Balance of supply treatment phase PBS restriction. Under no circumstances will approvals be granted for treatment that would otherwise extend the initial treatment period.

Where a response assessment is not conducted, the patient will be deemed to have not responded to treatment with this drug. A serious adverse reaction of a severity resulting in the necessity for permanent withdrawal of treatment is not considered as treatment failure.

Compliance with Authority Required procedures

C18993

Moderate to severe Crohn disease

Balance of supply

Patient must have received insufficient treatment with this drug for this condition under any relevant initial restriction (Initial 1, 2 or 3); AND

The treatment must provide no more than the balance of up to 14 weeks therapy available under Initial 1, 2 or 3 treatment.

Must be treated by a gastroenterologist; OR

Must be treated by a consultant physician [internal medicine specialising in gastroenterology]; OR

Must be treated by a consultant physician [general medicine specialising in gastroenterology]; OR

Must be treated by a paediatrician; OR

Must be treated by a specialist paediatric gastroenterologist.

Patient must be aged 6 to 17 years inclusive.

Compliance with Authority Required procedures

C18994

Moderate to severe Crohn disease

Initial 1 (new patient)

Patient must have confirmed diagnosis of Crohn disease, defined by standard clinical, endoscopic and/or imaging features including histological evidence; AND

Patient must not have achieved an adequate response to 2 of the following 3 conventional prior therapies including: (i) a tapered course of steroids, starting at a dose of at least 1 mg per kg or 40 mg (whichever is the lesser) prednisolone (or equivalent), over a 6 week period; (ii) an 8 week course of enteral nutrition; or (iii) immunosuppressive therapy with a thiopurine at a clinically optimised dose, or, methotrexate at a dose of at least 10 mg per square metre weekly for 3 or more months; OR

Patient must have a documented contraindication to each of prednisolone (or equivalent), a thiopurine, and methotrexate; OR

Patient must have features of high-risk or complicated Crohn disease defined as those with any of the following: (i) perianal disease, (ii) stricturing or penetrating behaviour, (iii) severe delayed growth (growth velocity at least 2 standard deviations below the mean for age and sex, or a drop of at least 1 major centile band); AND

Patient must have a Paediatric Crohn Disease Activity Index (PCDAI) Score greater than or equal to 30; OR

Patient must have extensive intestinal inflammation of the small intestine as evidenced by radiological imaging; AND

Patient must not receive more than 12-14 weeks of treatment under this restriction.

Must be treated by a gastroenterologist; OR

Must be treated by a consultant physician [internal medicine specialising in gastroenterology]; OR

Must be treated by a consultant physician [general medicine specialising in gastroenterology]; OR

Must be treated by a paediatrician; OR

Must be treated by a specialist paediatric gastroenterologist.

Patient must be aged 6 to 17 years inclusive.

The following information must be provided by the prescriber at the time of application and documented in the patient's medical records:

(a) if applicable, details of prior systemic drug therapy [dosage, date of commencement and duration of therapy]; and

(b) current PCDAI score and the date of assessment; or

(c) details (date, unique identifying number/code or provider number) of the pathology or diagnostic imaging test(s) used to assess response to therapy for patients with extensive small intestine disease.

For patients assessed as having extensive intestinal inflammation of the small intestines, such evidence of intestinal inflammation includes:

(i) blood: higher than normal platelet count, or, an elevated erythrocyte sedimentation rate (ESR) greater than 25 mm per hour, or, a C-reactive protein (CRP) level greater than 15 mg per L; or

(ii) faeces: higher than normal lactoferrin or calprotectin level; or

(iii) diagnostic imaging: demonstration of increased uptake of intravenous contrast with thickening of the bowel wall or mesenteric lymphadenopathy or fat streaking in the mesentery.

If treatment with any of the specified prior conventional drugs is contraindicated according to the relevant contraindications or precautions in the TGA-approved Product Information, please provide details at the time of application. Patients must attempt to trial alternate conventional therapies that are not contraindicated.

If intolerance to treatment develops during the relevant period of use, which is of a severity necessitating permanent treatment withdrawal, details of this toxicity must be provided at the time of application.

An increase in the maximum quantity to allow for dosing of up to 10 mg per kg body weight with 3 repeats will be authorised.

If fewer than 3 repeats are requested at the time of the application, authority approvals for sufficient repeats to complete the 3 doses of this drug may be requested through the Balance of supply treatment phase PBS restriction. Under no circumstances will approvals be granted for treatment that would otherwise extend the initial treatment period.

Where a response assessment is not conducted, the patient will be deemed to have not responded to treatment with this drug. A serious adverse reaction of a severity resulting in the necessity for permanent withdrawal of treatment is not considered as treatment failure.

Compliance with Authority Required procedures

C18995

Moderate to severe Crohn disease

Initial 2 (change or recommencement of treatment after a break in targeted therapy of less than 12 months)

Patient must have received prior PBS-subsidised treatment with a targeted therapy for this condition in this treatment cycle; OR

Patient must have been eligible for prior PBS-subsidised treatment with a targeted therapy for this condition in this treatment cycle; AND

Patient must not have previously received this drug for this condition in this treatment cycle; OR

Patient must have previously received this drug for this condition and demonstrated/maintained an adequate clinical response; OR

Patient must have: (i) received this drug in this treatment cycle, (ii) not demonstrated/maintained an adequate response to this drug once in this treatment cycle; AND

Patient must not receive more than 12-14 weeks of treatment under this restriction per authority application.

Must be treated by a gastroenterologist; OR

Must be treated by a consultant physician [internal medicine specialising in gastroenterology]; OR

Must be treated by a consultant physician [general medicine specialising in gastroenterology]; OR

Must be treated by a paediatrician; OR

Must be treated by a specialist paediatric gastroenterologist.

Patient must be aged 6 to 17 years inclusive.

The following information must be provided by the prescriber at the time of application and documented in the patient's medical records:

(a) if applicable, the PCDAI score and the date of assessment; or

(b) if applicable, details (date, unique identifying number/code or provider number) of the pathology or diagnostic imaging test(s) used to assess response to therapy for patients with extensive small intestine disease.

If a patient was eligible for prior PBS-subsidised treatment with a targeted therapy for this condition but did not receive PBS-subsidised treatment at the time, the application must provide a declaration that the patient met all 'Initial 1' clinical and treatment criteria when targeted therapy treatment commenced. The following information must also be provided at the time of application:

(a) the baseline PCDAI score and the date of assessment; or

(b) details (date, unique identifying number/code or provider number) of the pathology or diagnostic imaging test(s) used to assess response to therapy for patients with extensive small intestine disease.

An increase in the maximum quantity to allow for dosing of up to 10 mg per kg body weight with 3 repeats will be authorised.

If fewer than 3 repeats are requested at the time of the application, authority approvals for sufficient repeats to complete the 3 doses of this drug may be requested through the Balance of supply treatment phase PBS restriction. Under no circumstances will approvals be granted for treatment that would otherwise extend the initial treatment period.

Where a response assessment is not conducted, the patient will be deemed to have not responded to treatment with this drug. A serious adverse reaction of a severity resulting in the necessity for permanent withdrawal of treatment is not considered as treatment failure.

Compliance with Authority Required procedures

C19011

Moderate to severe Crohn disease

Continuing treatment

Patient must have received this drug as their most recent course of PBS-subsidised targeted therapy treatment for this condition; AND

Patient must have both: (i) a total PCDAI score of 30 points or less, and (ii) a reduction in PCDAI score by at least 15 points from baseline value; OR

Patient must have an adequate response to this drug defined as an improvement of intestinal inflammation as demonstrated by: (i) blood: normalisation of the platelet count, or an erythrocyte sedimentation rate (ESR) level no greater than 25 mm per hour, or a C-reactive protein (CRP) level no greater than 15 mg per L; or (ii) faeces: normalisation of lactoferrin or calprotectin level; or (iii) evidence of mucosal healing, as demonstrated by diagnostic imaging findings, compared to the baseline assessment; AND

Patient must not receive more than 24 weeks of treatment under this restriction per authority application.

Must be treated by a gastroenterologist; OR

Must be treated by a consultant physician [internal medicine specialising in gastroenterology]; OR

Must be treated by a consultant physician [general medicine specialising in gastroenterology]; OR

Must be treated by a paediatrician; OR

Must be treated by a specialist paediatric gastroenterologist.

Patient must be aged 6 to 17 years inclusive.

The measurement of response to the prior course of therapy must be documented in the patient's medical notes.

If a patient does not demonstrate a response to treatment with this drug they will not be eligible to receive further PBS-subsidised treatment with this drug for this condition under this treatment phase. Patients may re-trial treatment with this drug under the 'Initial 2' treatment phase if eligible. Serious adverse reaction of a severity resulting in the necessity for permanent withdrawal of treatment is not considered as a treatment failure.

An increase in the maximum quantity to allow for dosing of up to 10 mg per kg body weight will be authorised.

An increase in maximum repeats to 5 may be requested.

Compliance with Authority Required procedures - Streamlined Authority Code 19011

C19042

Moderate to severe Crohn disease

Continuing treatment

Patient must have received this drug as their most recent course of PBS-subsidised targeted therapy treatment for this condition; AND

Patient must have both: (i) a total PCDAI score of 30 points or less, and (ii) a reduction in PCDAI score by at least 15 points from baseline value; OR

Patient must have an adequate response to this drug defined as an improvement of intestinal inflammation as demonstrated by: (i) blood: normalisation of the platelet count, or an erythrocyte sedimentation rate (ESR) level no greater than 25 mm per hour, or a C-reactive protein (CRP) level no greater than 15 mg per L; or (ii) faeces: normalisation of lactoferrin or calprotectin level; or (iii) evidence of mucosal healing, as demonstrated by diagnostic imaging findings, compared to the baseline assessment; AND

Patient must not receive more than 24 weeks of treatment under this restriction per authority application.

Must be treated by a gastroenterologist; OR

Must be treated by a consultant physician [internal medicine specialising in gastroenterology]; OR

Must be treated by a consultant physician [general medicine specialising in gastroenterology]; OR

Must be treated by a paediatrician; OR

Must be treated by a specialist paediatric gastroenterologist.

Patient must be aged 6 to 17 years inclusive.

The measurement of response to the prior course of therapy must be documented in the patient's medical notes.

If a patient does not demonstrate a response to treatment with this drug they will not be eligible to receive further PBS-subsidised treatment with this drug for this condition under this treatment phase. Patients may re-trial treatment with this drug under the 'Initial 2' treatment phase if eligible. Serious adverse reaction of a severity resulting in the necessity for permanent withdrawal of treatment is not considered as a treatment failure.

An increase in the maximum quantity to allow for dosing of up to 10 mg per kg body weight will be authorised.

An increase in maximum repeats to 5 may be requested.

Compliance with Authority Required procedures - Streamlined Authority Code 19042

C18932

Relapsed or refractory follicular lymphoma

The treatment must be in combination with rituximab and tafasitamab; OR

The treatment must be in combination with tafasitamab.

The treatment must not exceed a total of 12 cycles in a lifetime for this indication measured from the initial dose, or must not extend beyond disease progression, whichever comes first.

Compliance with Authority Required procedures

Nitisinone

C18958

Hereditary tyrosinaemia type 1 (HT-1)

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 this drug; AND

The treatment must be in combination with dietary restriction of tyrosine and phenylalanine.

Must be treated by a physician with expertise in the management of HT-1; AND

Must be treated in a centre with expertise in genetic metabolic disorders.

At the time of authority application, the prescriber must request the appropriate quantity units of appropriate strength(s), based on the weight of the patient, to provide sufficient drug for up to 4 weeks of treatment at the dosage regimen specified in the approved Therapeutic Goods Administration (TGA) Product Information (PI). Up to 5 repeats will be authorised to provide for a total of 24 weeks of treatment.

A separate authority prescription form must be completed for each strength requested.

Liver function tests (LFT), Full blood count (FBC) and Alpha-fetoprotein (AFP) results confirming eligibility for continuing treatment must be documented in the patient's medical records.

Prescribers must state the patient's body weight with every authority application, and document it in the patient's medical records.

Compliance with Authority Required procedures

C18959

Hereditary tyrosinaemia type 1 (HT-1)

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 this drug; AND

The treatment must be in combination with dietary restriction of tyrosine and phenylalanine.

Must be treated by a physician with expertise in the management of HT-1; AND

Must be treated in a centre with expertise in genetic metabolic disorders.

Patient must be less than 18 years of age.

At the time of authority application, the prescriber must request the appropriate quantity units of appropriate strength(s), based on the weight of the patient, to provide sufficient drug for up to 4 weeks of treatment at the dosage regimen specified in the approved Therapeutic Goods Administration (TGA) Product Information (PI). Up to 5 repeats will be authorised to provide for a total of 24 weeks of treatment.

A separate authority prescription form must be completed for each strength requested.

Liver function tests (LFT), Full blood count (FBC) and Alpha-fetoprotein (AFP) results confirming eligibility for continuing treatment must be documented in the patient's medical records.

Prescribers must state the patient's body weight with every authority application, and document it in the patient's medical records.

Compliance with Authority Required procedures

C19053

Hereditary tyrosinaemia type 1 (HT-1)

Initial treatment

Patient must have a confirmed diagnosis of HT-1 based on the presence of succinylacetone in either: (i) urine, (ii) blood; AND

The treatment must be in combination with dietary restriction of tyrosine and phenylalanine; AND

Patient must not be suffering from any other severe or life-threatening medical condition, including complications or sequelae of HT-1, that might compromise the effectiveness of treatment with this drug or where the long-term prognosis is unlikely to be altered by treatment with this drug.

Must be treated by a physician with expertise in the management of HT-1; AND

Must be treated in a centre with expertise in genetic metabolic disorders.

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).

At the time of authority application, the prescriber must request the appropriate quantity units of appropriate strength(s), based on the weight of the patient, to provide sufficient drug for up to 4 weeks of treatment at the dosage regimen specified in the approved Therapeutic Goods Administration (TGA) Product Information (PI). Up to 5 repeats will be authorised to provide for a total of 24 weeks of treatment.

A separate authority prescription form must be completed for each strength requested.

Confirmation of eligibility for treatment with diagnostic reports including the blood and/or urine test results must be conducted no more than 12 months prior to the date of authority application and documented in the patient's medical records.

Prescribers must state the patient's body weight with every authority application, and document it in the patient's medical records.

Compliance with Authority Required procedures

C19055

Hereditary tyrosinaemia type 1 (HT-1)

Initial treatment

Patient must have a confirmed diagnosis of HT-1 based on the presence of succinylacetone in either: (i) urine, (ii) blood; AND

The treatment must be in combination with dietary restriction of tyrosine and phenylalanine; AND

Patient must not be suffering from any other severe or life-threatening medical condition, including complications or sequelae of HT-1, that might compromise the effectiveness of treatment with this drug or where the long-term prognosis is unlikely to be altered by treatment with this drug.

Must be treated by a physician with expertise in the management of HT-1; AND

Must be treated in a centre with expertise in genetic metabolic disorders.

Patient must be less than 18 years of age.

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).

At the time of authority application, the prescriber must request the appropriate quantity units of appropriate strength(s), based on the weight of the patient, to provide sufficient drug for up to 4 weeks of treatment at the dosage regimen specified in the approved Therapeutic Goods Administration (TGA) Product Information (PI). Up to 5 repeats will be authorised to provide for a total of 24 weeks of treatment.

A separate authority prescription form must be completed for each strength requested.

Confirmation of eligibility for treatment with diagnostic reports including the blood and/or urine test results must be conducted no more than 12 months prior to the date of authority application and documented in the patient's medical records.

Prescribers must state the patient's body weight with every authority application, and document it in the patient's medical records.

Compliance with Authority Required procedures

C19057

Hereditary tyrosinaemia type 1 (HT-1)

Grandfather arrangement (transition from LSDP-funded Hereditary Tyrosinaemia type 1 therapy)

Patient must have previously received treatment with this drug for this condition under the Australian Government's Life Saving Drugs Program (LSDP) prior to 1 August 2026; 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 this drug; AND

The treatment must be in combination with dietary restriction of tyrosine and phenylalanine.

Must be treated by a physician with expertise in the management of HT-1; AND

Must be treated in a centre with expertise in genetic metabolic disorders.

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).

At the time of authority application, the prescriber must request the appropriate quantity units of appropriate strength(s), based on the weight of the patient, to provide sufficient drug for up to 4 weeks of treatment at the dosage regimen specified in the approved Therapeutic Goods Administration (TGA) Product Information (PI). Up to 5 repeats will be authorised to provide for a total of 24 weeks of treatment.

A separate authority prescription form must be completed for each strength requested.

Confirmation of eligibility for treatment with diagnostic reports including the blood and/or urine test results at baseline, must be documented in the patient's medical records.

Prescribers must state the patient's body weight with every authority application, and document it in the patient's medical records.

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.

Compliance with Authority Required procedures

C19058

Hereditary tyrosinaemia type 1 (HT-1)

Grandfather arrangement (transition from LSDP-funded Hereditary Tyrosinaemia type 1 therapy)

Patient must have previously received treatment with this drug for this condition under the Australian Government's Life Saving Drugs Program (LSDP) prior to 1 August 2026; 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 this drug; AND

The treatment must be in combination with dietary restriction of tyrosine and phenylalanine.

Must be treated by a physician with expertise in the management of HT-1; AND

Must be treated in a centre with expertise in genetic metabolic disorders.

Patient must be less than 18 years of age.

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).

At the time of authority application, the prescriber must request the appropriate quantity units of appropriate strength(s), based on the weight of the patient, to provide sufficient drug for up to 4 weeks of treatment at the dosage regimen specified in the approved Therapeutic Goods Administration (TGA) Product Information (PI). Up to 5 repeats will be authorised to provide for a total of 24 weeks of treatment.

A separate authority prescription form must be completed for each strength requested.

Confirmation of eligibility for treatment with diagnostic reports including the blood and/or urine test results at baseline, must be documented in the patient's medical records.

Prescribers must state the patient's body weight with every authority application, and document it in the patient's medical records.

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.

Compliance with Authority Required procedures

Pegunigalsidase alfa

C18929

Fabry disease

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

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 August 2026; AND

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

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

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 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 authority application, prescribers must request the appropriate number of vials, based on the weight of the patient, to provide sufficient drug for 4 weeks of treatment at the dosage regimen specified in the approved Therapeutic Goods Administration (TGA) Product Information (PI).

Compliance with Written Authority Required procedures

C18930

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 this drug; AND

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

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

At the time of authority application, prescribers must request the appropriate number of vials, based on the weight of the patient, to provide sufficient drug for 4 weeks of treatment at the dosage regimen specified in the approved Therapeutic Goods Administration (TGA) Product Information (PI).

Compliance with Authority Required procedures

C19015

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 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

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

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

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.

At the time of authority application, prescribers must request the appropriate number of vials, based on the weight of the patient, to provide sufficient drug for 4 weeks of treatment at the dosage regimen specified in the approved Therapeutic Goods Administration (TGA) Product Information (PI).

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).

Compliance with Written Authority Required procedures

C19045

Fabry disease

Switching from PBS-subsidised migalastat

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

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

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

Patient must not have failed to demonstrate clinical improvement or stabilisation of this condition with previous PBS-subsidised treatment with this drug.

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

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

At the time of authority application, prescribers must request the appropriate number of vials, based on the weight of the patient, to provide sufficient drug for 4 weeks of treatment at the dosage regimen specified in the approved Therapeutic Goods Administration (TGA) Product Information (PI).

Compliance with Authority Required procedures

C18986

Type III Short bowel syndrome with intestinal failure

Initial treatment

Must be treated by a gastroenterologist; OR

Must be treated by a specialist within a multidisciplinary intestinal rehabilitation unit.

Patient must have short bowel syndrome with intestinal failure following major surgery; AND

Patient must have a history of dependence on parenteral support for at least 12 months; AND

Patient must have received a stable parenteral support regimen for at least 3 days per week in the 4 weeks prior to commencing this drug; AND

Patient must not have active gastrointestinal malignancy or history of gastrointestinal malignancy within the last 5 years; AND

The treatment must not exceed 12 months under this restriction; AND

Patient must not have previously received PBS-subsidised treatment with this drug for this condition.

Provide a baseline value in this authority application of the amount of parenteral support per week, expressed as either:

(i) for a patient of any age, the mean number of days of parenteral support per week,

(ii) for a patient yet to turn 18 years of age, the mean volume of parenteral support per week in mL per kg.

Determine the mean over any given 4 week period prior to commencing this drug. For a patient yet to turn 18 years of age, both (i) and (ii) may be supplied, but provide at least (i).

Assessment of treatment response/non-response in the 'Continuing treatment' authority application will be compared against the baseline value(s) submitted in this application.

A stable parenteral support regimen is defined as a minimum of 3 days of parenteral support (parenteral nutrition with or without IV fluids) per week for 4 consecutive weeks to meet caloric, fluid or electrolyte needs.

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

(a) details of the proposed prescription(s); and

(b) 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).

Compliance with Written Authority Required procedures