Gaucher patients in New Zealand are facing a change. New Zealand’s Pharmaceutical Management Agency (PHARMAC) recently proposed a change in their supported Gaucher treatment from Cerezyme to Elelyso. The switch is schedule to being in August, and within seventh months Elelyso would be the only PHARMAC funded enzyme replacement therapy for Gaucher. Keep reading to learn more, or follow the original story here for additional information.
What is Gaucher?
Gaucher disease is a lysosomal stage disorder. An enzyme responsible for the breakdown of certain compounds into glucose and ceramide either does not exist, or exists in incredibly low levels. Absent of this enzyme, certain compounds build up in the body which cause damage to tissues and organs. Gaucher affects about 1 in every 60,000 people. It is an autosomal recessive inherited disorder (meaning both parents must be carriers for a person to get Gaucher). The most common form of treatment for Gaucher is enzyme replacement therapy (ERT).
Why The Switch?
According to officials the switch from Cerezyme to Elelyso would expand access to an otherwise expensive treatment. It also helps the country to manage the costs of pharmaceuticals. With the proposal announced, PHARMAC requested feedback from Gaucher patients and their families.
20 patients have thus far been identified which are receiving Cerezyme. The proposal gives them until February of 2019 to change to Elelyso. Special requests and exceptions may be made for patients unable to tolerate Eleyso with approval from a physicians.
Patients currently being treated for Gaucher may need to undergo an MRI before switching to the new treatment. The first new treatment will also need to be administered in a hospital clinic. Future treatments can be administered by trained nurses at home. PHARMAC plans to assist hospital pharmacies during the transition, but local community pharmacies will need to work with clinicians during this period.
How to Be Involved
The new proposal details the ongoing actions of the Gaucher panel. The panel will be responsible for managing applications for the new treatment and renewing treatment for patients already receiving treatment. PHARMAC invites patients, and those close to patients to share their input on the new proposal. Opinions may be shared via email to [email protected].