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

Symptomatic obstructive hypertrophic cardiomyopathy

Initial treatment (covering the first 12 weeks of therapy)

Patient must have confirmed left ventricular hypertrophy due to hypertrophic cardiomyopathy; AND

Patient must have maximal end-diastolic left ventricular wall thickness which is at least one of either: (i) no less than 15 mm; (ii) no less than 13 mm if patient has familial hypertrophic cardiomyopathy (at least one first degree relative with a diagnosis of hypertrophic cardiomyopathy); AND

Patient must have confirmed peak left ventricular outflow tract (LVOT) gradient of no less than 50 mm Hg which is measured either: (i) at rest; (ii) after provocation with at least one of (a) Valsalva manoeuvre, (b) exercise; AND

Patient must have a current left ventricular ejection fraction (LVEF) of no less than 55%; AND

Patient must have had prior treatments with each of a (i) beta-blocker and (ii) non-dihydropyridine calcium channel blocker, unless at least one of the following is present: (a) a contraindication to beta-blocker and/or non-dihydropyridine calcium channel blocker therapy as listed in the TGA approved Product Information; (b) an intolerance to beta-blocker and/or non-dihydropyridine calcium channel blocker therapy; AND

Patient must be undergoing concomitant treatment with at least one of: (i) a beta-blocker (ii) non-dihydropyridine calcium channel blocker, unless at least one of the following is present: (a) a contraindication to beta-blocker and/or non-dihydropyridine calcium channel blocker therapy as listed in the TGA approved Product Information; (b) an intolerance to beta-blocker and/or non-dihydropyridine calcium channel blocker therapy; AND

Patient must be symptomatic with NYHA classes II or III; AND

Must be treated by a cardiologist; or

Must be treated by a consultant physician with experience in the management of hypertrophic cardiomyopathy; AND

Patient must be at least 18 years of age.

The authority application must be made in writing and must include all the following:

(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).

(3) The details of the echocardiogram and/ or cardiac magnetic resonance imaging (MRI) report confirming the diagnosis of hypertrophic cardiomyopathy (HCM). State all the following:

(a) the date, unique identifying number/code or provider number of the report;

(b) the left ventricular wall thickness in millimetres (mm).

(4) The details of a genotyping test report if the patient had been tested. State all the following:

(a) the date, unique identifying number/code or provider number of the report;

(b) if a gene has been identified that is associated with HCM;

(c) if any first-degree family relative has a confirmed diagnosis of HCM.

(5) The details of the LVOT gradient report. State all the following:

(a) the date, unique identifying number/code or provider number of the report;

(b) the measured LVOT gradient;

(c) how the LVOT gradient was measured (rest, Valsalva manoeuvre or exercise).

(6) NYHA status.

(7) The current beta-blocker or non-dihydropyridine calcium channel blocker (either diltiazem or verapamil only) therapy if applicable.

(8) Prior beta-blocker or non-dihydropyridine calcium channel blocker trials, including:

(a) if the patient is currently taking beta-blocker therapy, state the previous therapy with non-dihydropyridine calcium channel blocker that was trialled confirming that it was not effective;

(b) if the patient is currently taking non-dihydropyridine calcium channel blocker therapy, state the previous therapy with beta-blocker that was trialled confirming that it was not effective;

(c) if there is contraindication or intolerance to beta-blocker and/or non-dihydropyridine calcium channel blocker therapy as listed in the TGA approved Product Information, specify the details.

All results and reports must be documented in the patient's medical records.

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

Symptomatic obstructive hypertrophic cardiomyopathy

First continuing treatment (until at least 6 months on optimal dose is achieved)

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

Patient must be undergoing concomitant treatment with at least one of: (i) a beta-blocker (ii) non-dihydropyridine calcium channel blocker, unless at least one of the following is present: (a) a contraindication to beta-blocker and/or non-dihydropyridine calcium channel blocker therapy as listed in the TGA approved Product Information; (b) an intolerance to beta-blocker and/or non-dihydropyridine calcium channel blocker therapy; AND

Patient must have a current left ventricular ejection fraction (LVEF) of no less than 50%; AND

Patient must be titrating mavacamten treatment until optimal dose is achieved; or

Patient must be continuing mavacamten treatment to reach at least 6 months on the optimal dose prior to assessing the response; AND

Must be treated by a cardiologist; or

Must be treated by a consultant physician with experience in the management of hypertrophic cardiomyopathy.

The assessment of response must be conducted after at least 6 months on optimal dose to determine the patient's eligibility for maintenance treatment. Where an assessment is not undertaken, the patient will not be eligible for ongoing treatment. This treatment phase listing intends to provide up to 36 weeks of treatment in 3 treatment courses.

For the purposes of this restriction, an adequate response to treatment is defined as: an improvement in at least one of the following: (i) symptoms, (ii) quality of life, (iii) exercise capacity, (iv) peak left ventricular outflow tract (LVOT) gradient.