Decision to fund sapropterin

Medicines Decision

What we’re doing

We’re pleased to announce a decision to list sapropterin (Kuvan), a treatment for the rare condition phenylketonuria (PKU), for women who are pregnant or actively planning a pregnancy and require pharmacological support to manage PKU in pregnancy.

Sapropterin will be funded from 1 November 2018, through an agreement with BioMarin Pharmaceutical Australia Pty Ltd.

Any changes to the original proposal?

Following consideration of consultation responses, we approved the terms of listing outlined in our consultation letter dated 30 August 2018 without any changes.

Who we think will be most interested

  • Women with PKU who are pregnant or planning a pregnancy.
  • PKU consumer interest groups.
  • Health practitioners involved in the management of patients with PKU.
  • Hospital and community pharmacists, DHBs, suppliers and wholesalers.

Detail about this decision

Sapropterin (Kuvan) will be listed in Section B and Part II of Section H of the Pharmaceutical Schedule from 1 November 2018 at the following price and subsidy (ex-manufacturer, excluding GST):

Chemical

Presentation

Brand

Pack size

Price and subsidy

Sapropterin dihydrochloride

Tab soluble 100 mg

Kuvan

30 OP

$1,452.70

A confidential rebate will apply to Kuvan that will reduce its net price to the Funder.

Kuvan will have subsidy and delisting protection until 31 October 2019.

Original (whole) pack (OP) dispensing will apply. Kuvan is supplied in bottles of 30 tablets, which should be used within two months after first opening.

Sapropterin will be listed in Section B of the Pharmaceutical Schedule subject to the following Special Authority criteria:

Special Authority for Subsidy

Initial application only from a metabolic physician. Approvals valid for 1 month for applications meeting the following criteria:

All of the following:

  1. Patient has phenylketonuria (PKU) and is pregnant or actively planning to become pregnant; and
  2. Treatment with sapropterin is required to support management of PKU during pregnancy; and
  3. Sapropterin to be administered at doses no greater than a total daily dose of 20 mg/kg; and
  4. Sapropterin to be used alone or in combination with PKU dietary management; and
  5. Total treatment duration with sapropterin will not exceed 22 months for each pregnancy (includes time for planning and becoming pregnant) and treatment will be stopped after delivery.

Renewal only from a metabolic physician or relevant practitioner on the recommendation of a metabolic physician. Approvals valid for 12 months for applications meeting the following criteria:

All of the following:

  1. Either:

1.1     Following the initial one-month approval, the patient has demonstrated an adequate response to a 2 to 4 week trial of sapropterin with a clinically appropriate reduction in phenylalanine levels to support management of PKU during pregnancy; or
1.2     On subsequent renewal applications, the patient has previously demonstrated response to treatment with sapropterin and maintained adequate phenylalanine levels to support management of PKU during pregnancy; and

  1. Any of the following:

2.1     Patient continues to be pregnant and treatment with sapropterin will not continue after delivery; or
2.2     Patient is actively planning a pregnancy and this is the first renewal for treatment with sapropterin; or
2.3     Treatment with sapropterin is required for a second or subsequent pregnancy to support management of their PKU during pregnancy; and

  1. Sapropterin to be administered at doses no greater than a total daily dose of 20 mg/kg; and  
  2. Sapropterin to be used alone or in combination with PKU dietary management; and
  3. Total treatment duration with sapropterin will not exceed 22 months for each pregnancy (includes time for planning and becoming pregnant) and treatment will be stopped after delivery.

Similar restrictions will apply in Part II of Section H of the Pharmaceutical Schedule (the Hospital Medicines List; HML).

Our response to what you told us

We appreciate the time people took to respond to this consultation. All responders supported the funding of sapropterin for use in pregnancy by women with PKU. A summary of the main theme raised in feedback and our response to this is shown below.

Theme

PHARMAC response

Concerns about prescriber type restriction in the Special Authority criteria:

  
  1. Obstetricians will be involved in the care of pregnant women with PKU at CDHB.

The Special Authority criteria were developed in consultation with clinicians involved in the care of patients with PKU.
  1. ‘Metabolic Physician’ is not a registered specialty.

The term ‘Metabolic Physician’ is consistent with Special Authority criteria for other medicines used for metabolic disorders and funded by PHARMAC. This term also maps to clinical geneticist and paediatrician specialties in the Special Authority application approval system.
  1. Requested that PHARMAC uses recognised and registered specialties when describing prescriber restrictions.

We understand that these patients may be under the care of a range of health practitioners and other specialists (including obstetricians and dietitians). The renewal criteria include “on the recommendation of a metabolic physician” in recognition that, following the initial trial to confirm response to sapropterin, other clinicians will be involved in the care of women with PKU who are, or are planning to become, pregnant.

If you have any questions about this decision, you can email us at enquiry@pharmac.govt.nz; or call our toll free number (9 am to 5 pm, Monday to Friday) on 0800 66 00 50.