Blog
RSS FeedPosted by Michael Wonder on 18 Nov 2019
Funding orphan medicinal products beyond price: sustaining an ecosystem
18 November 2019 - Following a similar legislation in the USA, the EU passed a legislation in 1999 to incentivise ...
Posted by Michael Wonder on 12 Nov 2019
Rare Disease Fund now covers Pompe disease, a rare inherited neuromuscular disorder
3 November 2019 - The Rare Disease Fund now covers Singaporeans with Pompe disease - a rare inherited neuromuscular disorder ...
Posted by Michael Wonder on 11 Nov 2019
FDA admits it goofed when granting orphan status to an opioid addiction treatment
8 November 2019 - In an unusual move, the FDA has acknowledged a mistake and revoked orphan drug status for ...
Posted by Michael Wonder on 01 Nov 2019
Vertex announces EMA marketing authorisation application validation for VX-445 (elexacaftor), tezacaftor and ivacaftor triple combination treatment in cystic fibrosis
31 October 2019 - Application supported by positive results from two global Phase 3 studies in people with cystic fibrosis ages ...
Posted by Michael Wonder on 29 Oct 2019
Battle for life-saving drugs now harder as PHARMAC absorbs $5m rare disorders fund
29 October 2019 - A $5 million fund set up by PHARMAC in 2014 to address rare disorders no longer ...
Posted by Michael Wonder on 24 Oct 2019
NHSE signs ‘definitive’ agreement for Vertex CF drugs
24 October 2019 - NHS England has announced a definitive agreement with Vertex Pharmaceuticals to make all three of their ...
Posted by Michael Wonder on 08 Oct 2019
FDA awards 12 grants to fund new clinical trials to advance the development of medical products for the treatment of rare diseases
8 October 2019 - The U.S. FDA today announced that it has awarded 12 new clinical trial research grants totaling ...
Posted by Michael Wonder on 06 Oct 2019
Mum 'hopeful' after baby Kaan Hunter starts $250k a year medication for rare bone disease
5 October 2019 - A mum whose baby was the first Kiwi to be diagnosed with a rare bone disease ...
Posted by Michael Wonder on 17 Sep 2019
News NHS England explores funding options for cystic fibrosis drug
17 September 2019 - NHS England is “committed to exploring and developing alternative routes” to enable people with cystic fibrosis ...
Posted by Michael Wonder on 13 Sep 2019
Vertex announces new access agreement with Scottish Government for Orkambi (lumacaftor/ivacaftor) and Symkevi (tezacaftor/ivacaftor)
12 September 2019 - Eligible patients in Scotland will immediately have access to Orkambi (lumacaftor/ivacaftor) and Symkevi (tezacaftor/ivacaftor) in combination with ...
Posted by Michael Wonder on 12 Sep 2019
Batten disease treatment: parents win battle ahead of court date
11 September 2019 - Families of children with a rare degenerative disease have won their fight for funding for NHS ...
Posted by Michael Wonder on 11 Sep 2019
Children with rare inherited condition to benefit from drug through managed access agreement
11 September 2019 - It is estimated that in the UK there are around 30 to 50 children living with the ...
Posted by Michael Wonder on 03 Sep 2019
The FDA and Sarepta: a window into the real world of drug regulation
3 September 2019 - It is hard to discern the true state of drug regulation from the outside, but two ...
Posted by Michael Wonder on 28 Aug 2019
European Commission approves Soliris (eculizumab) for the treatment of adults with neuromyelitis optica spectrum disorder
27 August 2019 - 98% of adult anti-aquaporin-4 antibody-positive patients treated with Soliris were relapse free compared to 63% receiving placebo ...
Posted by Michael Wonder on 20 Aug 2019
FDA accepts new drug application for VX-445 (elexacaftor), tezacaftor and ivacaftor combination treatment
20 August 2019 - FDA grants priority review of the application and sets a PDUFA target action date of 19 ...