![]() That's an important piece in terms of equity. I'd like to, on behalf of all the families, thank the Minister and the Australian Government for making the very foresighted decision to list this new medication, Voxzogo, on the PBS scheme, which means that for every child in Australia, and for every parent in Australia who wishes to use this medication as a lifesaving and definitely lifechanging medication in their child, they have the opportunity to do so. It's an absolute pleasure to be here and today marks a very special occasion. PROFESSOR RAVI SAVAIRAYAN: Thank you very much, Minister. Without further ado, I'll ask Professor Ravi to say a few words and then after a couple of more contributions, I'm happy to take questions. As I've said, amazing work now pays off for the recognition of this treatment, and a listing on the PBS and obviously also the pharmaceutical company that has led this work BioMarin. I want to pay amazing tribute to the families who've been part of these clinical trials, the researchers from the Murdoch Children’s Research Institute that have led the clinical trials work around the world here for 10 years. Instead of paying $330,000, this treatment will be available at PBS prices – $30 a script or even $7 for concessional patients. So, $330,000 a year for maybe 15 to 16 years of life is obviously beyond the means of any family, which is why the PBS is such a terrific medicine system here in Australia. These treatments will start very young in life, Dr Ravi will talk about that, but very, very young and endure right through until the growth plates close, which is around 16 or 18 years of age. Without a PBS listing, this treatment would cost as much as $330,000 for families every single year. We think there are about 140 young Australians who will benefit from this listing every single year and being listed on the PBS, as people know, makes it affordable for families. Those clinical trials over the last 10 years have shown terrific effects for these young children, and has now been approved by the Pharmaceutical Benefits Advisory Committee and listed on the PBS. Now, here in Australia, led by the Murdoch Children’s Research Institute, particularly Professor Ravi who will say a few words, there have been clinical trials underway with the company BioMarin, for a new treatment that effectively attacks the genetic causes of this condition and allows the bone growth to resume in a much more standard way. And if they survive that long, there are a range of very invasive major pieces of surgery, they are likely to have to undertake, as well. Patients with this condition are 50 times more likely to die before their fifth birthday than the national average. This is a very serious condition that particularly impacts people when they're at their younger ages. ![]() ![]() ![]() This is a terrific, exciting new treatment for achondroplasia, which is the most common form of dwarfism all around the world. Today, I'm delighted to announce that Voxzogo will be listed on the PBS, effective from the 1st of May. Truly nation leading work, and world leading work undertaken here over the last 10 years in Australia. MINISTER FOR HEALTH AND AGED CARE MARK BUTLER: Thanks so much for coming out for this really, really exciting occasion for the listing of a terrific new treatment – a world leading new hope for life for hundreds and hundreds of families here in Australia but around the world as well.
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