Posted by Michael Wonder on 17 May 2018
Proposal to widen access and change the funded enzyme replacement therapy for Gaucher disease
17 May 2018 - PHARMAC is seeking feedback on proposed changes to the funding of enzyme replacement therapy used in the treatment of Gaucher disease.
- The funded enzyme replacement therapy for the treatment of Gaucher disease would change from imiglucerase (Cerezyme), supplied by Sanofi, to taliglucerase alfa (Elelyso), supplied by Pfizer.
- Taliglucerase alfa would be funded from 1 August 2018 and would become the only funded ERT for the treatment of Gaucher disease in the community after a 7-month transition period.
- Special Authority and Hospital Restrictions would apply to taliglucerase alfa. These would be similar to the restrictions that currently apply to imiglucerase, except that, if approved by the Gaucher Panel (a panel of expert doctors), the criteria would allow a higher maximum dose (30 units/kg every other week, approx. 60 units/kg/month) where clinically appropriate, which could provide clinical benefits for some patients.
