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

Spinal muscular atrophy (SMA)

Initial treatment occurring after onasemnogene abeparvovec therapy in a patient with Type 1 SMA

Patient must have experienced a regression in a developmental state listed below (see 'Definition') despite treatment with gene therapy - confirm that this: (i) not due to an acute concomitant illness; (ii) not due to non-compliance to best-supportive care, (iii) apparent for at least 3 months, (iv) verified by another clinician in the treatment team - state the full name of this clinician plus their profession (e.g. medical practitioner, nurse, physiotherapist; this is not an exhaustive list of examples); AND

The treatment must not be a PBS-subsidised benefit where the condition has progressed to a point where invasive permanent assisted ventilation (i.e. ventilation via tracheostomy tube for at least 16 hours per day) is required in the absence of potentially reversible causes; AND

The treatment must be given concomitantly with best supportive care for this condition; AND

The treatment must not be in combination with PBS-subsidised treatment with nusinersen for this condition; AND

Must be treated by a specialist medical practitioner experienced in the diagnosis and management of SMA associated with a neuromuscular clinic, or in consultation with a specialist medical practitioner experienced in the diagnosis and management of SMA associated with a neuromuscular clinic; AND

Patient must be undergoing treatment under this Treatment phase listing once only - for continuing treatment beyond this authority application, refer to the drug's relevant 'Continuing treatment' listing for the patient's SMA type; AND

Patient must have a prior authority approval for any drug PBS-listed for symptomatic Type 1 SMA, with at least one approval having been for gene therapy.

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

(1) details of the proposed prescription; 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).

Do not resubmit previously submitted documentation concerning the diagnosis and type of SMA.

Confirm that a previous PBS authority application has been approved for symptomatic Type 1 SMA.

Definition:

Various childhood developmental states (1 to 9) are listed below, some followed by further observations (a up to d). Where at least one developmental state/observation is no longer present, that developmental state has regressed.

0. Absence of developmental states (1 to 9) listed below:

1. Rolls from side to side on back;

2. Child holds head erect for at least 3 seconds unsupported;

3. Sitting, but with assistance;

4. Sitting without assistance:

(a) Child sits up straight with the head erect for at least 10 seconds;

(b) Child does not use arms or hands to balance body or support position.

5. Hands and knees crawling:

(a) Child alternately moves forward or backwards on hands and knees;

(b) The stomach does not touch the supporting surface;

(c) There are continuous and consecutive movements at least 3 in a row.

6. Standing with assistance:

(a) Child stands in upright position on both feet, holding onto a stable object (e.g. furniture) with both hands and without leaning on object;

(b)The body does not touch the stable object, and the legs support most of the body weight;

(c) Child thus stands with assistance for at least 10 seconds.

7. Standing alone:

(a) Child stands in upright position on both feet (not on the toes) with the back straight;

(b) The leg supports 100% of the child's weight;

(c) There is no contact with a person or object;

(d) Child stands alone for at least 10 seconds.

8. Walking with assistance:

(a) Child is in an upright position with the back straight;

(b) Child makes sideways or forced steps by holding onto a stable object (e.g. furniture) with 1 or both hands;

(c) One leg moves forward while the other supports part of the body weight;

(d) Child takes at least 5 steps in this manner.

9. Walking alone:

(a) Child takes at least 5 steps independently in upright position with the back straight;

(b) One leg moves forward while the other supports most of the body weight;

(c) There is no contact with a person or object.

Confirm which developmental state has regressed by: (i) stating the overall developmental state (1 - 9) the patient was in at the time of gene therapy, or, the best developmental state achieved since gene therapy, and (ii) stating the patient's current overall developmental state (i.e. a number that is lower than stated in (i).

Where the patient has neither regressed from a developmental state nor reached the next developmental state, PBS-subsidy of this benefit is not available.

The approved Product Information recommended dosing is as follows:

(i) 16 days to less than 2 months of age: 0.15 mg/kg

(ii) 2 months to less than 2 years of age: 0.20 mg/kg

(iii) 2 years of age and older weighing less than 20 kg: 0.25 mg/kg

(iv) 2 years of age and older weighing 20 kg or more: 5 mg

In this authority application, state which of (i) to (iv) above applies to the patient. Based on (i) to (iv), prescribe up to:

1 unit where (i) applies;

2 units where (ii) applies;

3 units where (iii) applies;

3 units where (iv) applies.