Mindarie family joins call for drug to treat son’s condition be placed on ‘life saving drugs program’


Coby Antonio. Picture: Martin Kennealey
Coby Antonio. Picture: Martin Kennealey

ADVOCATES are raising awareness of rare diseases in a bid to make treatments more accessible to those who need it.

On Rare Disease Day, a Mindarie family joined the call for the Federal Government to add the drug Vimizim to the ‘Life saving drugs program’.

Coby Antonio (15) has a rare condition, Morquio A, and has undergone six knee operations to correct his malformed joints.

Born eight weeks premature, Coby was diagnosed with the ‘knock knee’ condition as a three-year-old after his mother Sonia noticed he could not cross his legs.

Every week for more than three and a half years, they have travelled to Princess Margaret Hospital so Coby can receive enzyme replacement therapy (ERT).

The treatment is not available for government subsidy and has been given to Coby compassionately through the manufacturer.

Mrs Antonio said his stamina, weight and muscle tone had improved since he started the treatment and he could now participate in physical activity.

She said access to ERT meant they were no longer reactively just fixing problems that come about and she hoped it would be made publicly available to other people living with Morquio A.

“(Their) little bodies are working against them, and they do not have a treatment option,” she said.

The Pharmaceutical Benefits Advisory Committee (PBAC) rejected Vimizim, or elosulfase alfa, for Morquio A, for funding on the Pharmaceutical Benefits Scheme (PBS) on cost effectiveness grounds at its March 2016 meeting.

The committee did accept its clinical effectiveness, saying that Vimizim was “superior to placebo in terms of efficacy but associated with a slightly greater incidence of adverse events”.

Made publicly available on April 22, 2016, the decision was sent to the chief medical officer to provide advice to the Minister for Health on potentially including Vimizim on the ‘Life saving drugs program’ but advocates say a decision is yet to be made.

A Federal Health Department spokeswoman said Australia’s Chief Medical Officer, Brendan Murphy, was currently assessing a proposal for the subsidy of this medicine under the Life Saving Drug Program, which the department received on February 23.

She said the independent PBAC had considered the drug for the PBS on two occasions, with the most recent in March 2016.

“The sponsor of the medicine, BioMarin, is free to make a resubmission to the PBAC at any time, although no resubmission has been made since March 2016,” the spokeswoman said.

The Vimizim website carries a warning about potential side effects of the treatment.

“Life-threatening allergic reactions have occurred in some patients during Vimizim (elosulfase alfa) infusions and up to three hours after infusion,” the website said.

“Patients with acute respiratory illness may be at increased risk and require additional monitoring.”