My son Kalten lives with the ultra-rare and debilitating disorder Morquio A or MPS IV.
This week we celebrated his fourth birthday. While birthdays are a happy occasion, my family were hoping that we would have much more to celebrate this year. We were hopeful to receive news that the first and only treatment available for Australians with Morquio A, Vimizim® (elosulfase alfa), would be reimbursed by the Federal Government. Sadly, this wasn’t the case – and we are no closer to knowing whether Kalten, and other patients with Morquio A, will be given access to this treatment.
There are around 21 Australians with Morquio A, a genetic condition that affects major organs in the body. People born with Morquio A are missing, or don’t make enough of, an important enzyme and can’t break down certain materials in the body. This causes a number of serious health problems, with most children having to undergo dozens of surgeries before they reach the age of five.
Kalten was diagnosed with Morquio A in January 2015, just months after the Australian clinical trial of Vimizim closed. This meant we had no option but to wait for the Government to decide to fund Vimizim so that Kalten, and other patients who are newly diagnosed, can access the treatment. Besides Kalten, there are two other children who are also missing out on treatment because their diagnosis came after the clinical trial had closed.
About 13 Australians were able to access the clinical trial - including Beau Cosgrove, one of Kalten’s friends. It’s amazing to see how much Beau’s physical health and general wellbeing have improved since being on the treatment. He is able to move better, walk without help, breathe normally and start school – the improvement to his quality of life has been a joy for his family to see.
The international treatment guidelines for Morquio A say that treatment with Vimizim should be started as early as possible after a child is diagnosed, in order to maximise the possible benefits. It has now been 18 months since Kalten’s diagnosis and yet; he still has been unable to access the treatment that will improve his life.
Vimizim was considered by the Pharmaceutical Benefits Advisory Committee (PBAC) at its March 2016 meeting. The PBAC stated that the treatment cost too much, but accepted its clinical effectiveness. This outcome was made public in April.
Since then, we – along with other Morquio A families and the broader MPS community – have been left in the dark with no further update on the status of the submission. Kalten, and the other two Morquio A children without access to treatment, are now being forced to wait even longer for an outcome, while their conditions continue to worsen. This should never have been a situation my son had to face, but because of delays to the process, all we can do is hope that a decision known soon.