Blog
RSS FeedPosted by Michael Wonder on 10 Nov 2020
Mirum Pharmaceuticals broadens Expanded Access Program for maralixibat in Alagille syndrome to Europe and Australia
5 November 2020 - Maralixibat Expanded Access Program now available for patients with pruritus associated with Alagille syndrome in Australia and ...
Posted by Michael Wonder on 12 Mar 2019
Vertex receives approval for Symdeko (tezacaftor/ivacaftor and ivacaftor) in Australia, to treat the underlying cause of cystic fibrosis in people aged 12 and older with certain CFTR gene mutations
12 March 2019 - A new treatment option for patients with two copies of the F508del mutation, the most common mutation ...
Posted by Michael Wonder on 12 Jul 2018
Two boys, one horrific disease, different wonder drug funding
12 July 2018 - A young boy dying of a rare and horrifying genetic disease faces losing access to a ...
Posted by Michael Wonder on 07 Jun 2018
Reform of the orphan drug program - End of transition arrangements
7 June 2018 - On 1 July 2017 amendments to the Therapeutic Goods Regulations 1990 (the Regulations) came into effect that ...
Posted by Michael Wonder on 05 Feb 2018
Cancer research funding has an inherent bias that often excludes rare cancers, expert says
3 February 2018 - The biggest investor in cancer research in Australia needs to address an "inherent bias" in how ...
Posted by Michael Wonder on 16 Aug 2017
Amicus Therapeutics announces approval for Galafold (migalastat) for treatment of Fabry disease in Australia
15 August 2017 - First Amicus medicine and first oral precision medicine for Fabry disease in Australia. ...
Posted by Michael Wonder on 26 Jun 2017
Orphan drug program reforms
26 June 2017 - The objective of the orphan drug program is to provide an incentive to sponsors to bring ...
Posted by Michael Wonder on 01 May 2017
Reform of the Orphan Drug Program - transition arrangements
1 May 2017 - The Orphan Drug Program provides an incentive for sponsors to bring medicines for a small population to ...
Posted by Michael Wonder on 18 Apr 2017
Submissions received and TGA response: orphan drug program
18 April 2017 - The TGA would like to thank respondents who provided submissions in response to the October 2016 ...
Posted by Michael Wonder on 21 Sep 2016
TGA publishes AusPAR for Orkambi
21 September 2016 - The TGA has published an AusPAR for Vertex's combination product for cystic fibrosis. ...
Posted by Michael Wonder on 05 Jul 2016
TGA publishes AusPAR for asfotase alfa (Strensiq)
5 July 2016 - The TGA has published an AusPAR for Alexion Pharmaceuticals' Strensiq. ...
Posted by Michael Wonder on 09 Mar 2016
Vertex receives Australian approval for Orkambi (lumacaftor/ivacaftor), the first medicine to treat the underlying cause of cystic fibrosis in people ages 12 and older with two copies of the F508del mutation
8 March 2016 - The Australian reimbursement process for Orkambi is already underway as part of the parallel regulatory and reimbursement ...