Proposal to list galsulfase (Naglazyme) for mucopolysaccharidosis (MPS) VI
3 February 2016
PHARMAC is seeking feedback on a proposal to list galsulfase (Naglazyme) resulting from a provisional agreement between BioMarin Pharmaceutical Australia Pty Ltd and PHARMAC.
The provisional agreement is the third that PHARMAC has reached with a bidder from a Request for Proposals we ran in 2014, related to the supply of medicines for rare disorders.
In summary, this proposal would result in galsulfase (Naglazyme) being funded in the community under Special Authority criteria and in DHB hospitals subject to restrictions. Listing in the Pharmaceutical Schedule would occur subject to Medsafe approval of the pharmaceutical.
Galsulfase is an enzyme replacement therapy administered as an intravenous infusion for the treatment of the rare disorder Maroteaux-Lamy Syndrome (mucopolysaccharidosis (MPS) VI).
Detail of the proposal and some background information is set out below.
PHARMAC welcomes feedback on this proposal. To provide feedback, please submit it in writing by 5pm on Thursday, 18 February 2016 to:
Caroline De Luca
Senior Therapeutic Group Manager/Team Leader
PO Box 10 254
Fax: 04 460 4995
All feedback received before the closing date will be considered by PHARMAC’s Board (or its delegate) prior to making a decision on this proposal.
Feedback we receive is subject to the Official Information Act 1982 (OIA) and we will consider any request to have information withheld in accordance with our obligations under the OIA. Anyone providing feedback, whether on their own account or on behalf of an organisation, and whether in a personal or professional capacity, should be aware that the content of their feedback and their identity may need to be disclosed in response to an OIA request.
We are not able to treat any part of your feedback as confidential unless you specifically request that we do, and then only to the extent permissible under the OIA and other relevant laws and requirements. If you would like us to withhold any commercially sensitive, confidential proprietary, or personal information included in your submission, please clearly state this in your submission and identify the relevant sections of your submission that you would like it withheld. PHARMAC will give due consideration to any such request.
Details of the proposal
Following Medsafe approval, galsulfase (Naglazyme) would be listed in Section B (the Community) and Part II of Section H (the Hospital Medicines List) of the Pharmaceutical Schedule as follows:
Price and subsidy
Inj 1 mg per ml, 5 ml vial
- A confidential rebate would apply to Naglazyme, which would reduce the net price of the treatment to the Funder.
- Naglazyme would be listed as soon as practicable following BioMarin’s notification to PHARMAC that Medsafe has granted registration.
- Naglazyme would be listed subject to the following Special Authority criteria in the community and equivalent restrictions in the Hospital Medicines List:
Special Authority for Subsidy
Initial application only from a metabolic physician. Approvals valid for 12 months for patients meeting the following criteria:
- The patient has been diagnosed with mucopolysaccharidosis VI; and
2.1. Diagnosis confirmed by demonstration of N-acetyl-galactosamine-4-sulfatase (arylsulfatase B) deficiency by either enzyme activity assay in leukocytes or skin fibroblasts; or
2.2. Detection of two disease causing mutations and patient has a sibling who is known to have mucopolysaccharidosis VI.
Renewal only from a metabolic physician. Approvals valid for 12 months for applications meeting the following criteria:
All of the following:
- The treatment remains appropriate for the patient and the patient is benefiting from treatment; and
- Patient has not had severe infusion-related adverse reactions which were not preventable by appropriate pre-medication and/or adjustment of infusion rates; and
- Patient has not developed another life threatening or severe disease where the long term prognosis is unlikely to be influenced by Enzyme Replacement Therapy (ERT); and
- Patient has not developed another medical condition that might reasonably be expected to compromise a response to ERT.
- Naglazyme would have Sole Supply Status in the community, Hospital Supply Status in DHB hospitals and subsidy and delisting protection until 30 June 2018.
- Prior to a listing on the Pharmaceutical Schedule (ie until Medsafe approval is granted) net pricing would apply to any individual patient funding applications approved via the Named Patient Pharmaceutical Assessment (NPPA) Policy.
Maroteaux-Lamy Syndrome (mucopolysaccharidosis (MPS) VI) is rare genetic lysosomal storage disorder that usually presents in childhood and involves bones, joints, eyes, heart valves, causes hepatosplenomegaly, and affects the nervous system. It varies in severity between patients, with some patients having few manifestations whereas others may have life threatening complications and severely reduced quality of life. MPS VI is caused by deficient activity of an enzyme (N-acetylgalactosamine 4-sulfatase) that breaks down excessive glycosaminoglycan (GAG) that has built up in cells. Accumulation of GAG leads to progressive organ damage and to MPS VI symptoms.
PHARMAC estimates there to be currently fewer than 5 patients in New Zealand with MPS VI. There are currently no funded alternative treatments for MPS VI.
Galsulfase (Naglazyme) is a recombinant enzyme replacement therapy (ERT) that increases the breakdown of GAG. It is administered as an intravenous infusion once weekly. Treatment may improve mobility, exercise tolerance, and preserve respiratory and cardiac function.
Naglazyme is approved in Europe, US, Australia and other countries. BioMarin has submitted an application for registration to Medsafe, and it is currently under assessment (see http://www.medsafe.govt.nz/regulatory/ProductDetail.asp?ID=17616(external link)).
PHARMAC has not received any NPPA applications for the funding of galsulfase for individuals.
PHARMAC’s evaluation of galsulfase (Naglazyme) followed the process described in Schedule 2 of the Request for Proposals (RFP) for the supply of medicines for rare disorders. The RFP document can be found on the PHARMAC website.
Last updated: 27 June 2018