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

Achondroplasia

Initial treatment

Patient must have a diagnosis of achondroplasia, confirmed by appropriate genetic testing; AND

Patient must not have evidence of growth plate closure demonstrated by at least one of the following: i) bilateral lower extremity X-rays (proximal tibia, distal femur) taken within 6 months of this application if puberty has commenced; ii) bilateral lower extremity X-rays (proximal tibia, distal femur) taken within 2 years of commencing treatment if puberty has not commenced; iii) an annual growth velocity of greater than 1.5 cm/year as assessed over a period of at least 6 months; AND

Must be treated by a medical specialist, experienced in the management of achondroplasia; or

Must be treated by a paediatrician in consultation with a medical specialist experienced in the management of achondroplasia.

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.

Appropriate genetic testing constitutes testing for FGFR3 gene mutation.

In patients where puberty has not commenced, radiographic evidence that epiphyses have not closed must be obtained within 2 years of commencing treatment with vosoritide. X-rays and dates (date commenced treatment and date of X-ray) must be documented in the patient's medical records.

Additional radiographic evidence is not required until patient has begun puberty.

In patients where puberty has commenced, radiographic evidence that epiphyses have not closed must be obtained within 6 months of completing an authority application for vosoritide. X-ray and date taken must be documented in the patient's medical records.

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

Achondroplasia

Continuing treatment

Patient must have received PBS subsidised vosoritide treatment for this condition; AND

Patient must not have evidence of growth plate closure demonstrated by at least one of the following: i) bilateral lower extremity X-rays (proximal tibia, distal femur) taken within 6 months of this application if puberty has commenced; ii) bilateral lower extremity X-rays (proximal tibia, distal femur) taken within 2 years of commencing treatment if puberty has not commenced; iii) an annual growth velocity of greater than 1.5 cm/year as assessed over a period of at least 6 months; AND

Must be treated by a medical specialist, experienced in the management of achondroplasia; or

Must be treated by a paediatrician in consultation with a medical specialist experienced in the management of achondroplasia.

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.

In patients where puberty has not commenced, radiographic evidence that epiphyses have not closed must be obtained within 2 years of commencing treatment with vosoritide. X-rays and dates (date commenced treatment and date of X-ray) must be documented in the patient's medical records.

Additional radiographic evidence is not required until patient has begun puberty.

In patients where puberty has commenced, radiographic evidence that epiphyses have not closed must be obtained within 6 months of completing an authority application for vosoritide. X-ray and date taken must be documented in the patient's medical records.