Blog
RSS FeedPosted by Michael Wonder on 16 May 2019
Mum delivers 65 roses to Downing Street in bid to speed up NHS availability of vital cystic fibrosis drugs
16 May 2019 - Calls for NHS access to potentially life-saving drugs are growing, one year to the day after ...
Posted by Michael Wonder on 13 May 2019
MPs call for 'life-changing' Kuvan to be made affordable
11 May 2019 - A number of MPs are calling on a drug company to make a "life-changing" treatment affordable ...
Posted by Michael Wonder on 08 May 2019
FDA undercuts $375,000 drug in surprise move
8 May 2019 - The US FDA created a workaround this week that effectively undercuts the $375,000 price tag of ...
Posted by Michael Wonder on 08 May 2019
Akcea and Ionis announce approval of Waylivra (volanesorsen) in the European Union
7 May 2019 - Akcea Therapeutics and Ionis Pharmaceuticals announced today that Waylivra has received conditional marketing authorisation from the ...
Posted by Michael Wonder on 07 May 2019
European Commission approves Palynziq (pegvaliase injection) for treatment of phenylketonuria in patients aged 16 years or older
6 May 2019 - First enzyme substitution therapy approved in Europe to treat the underlying cause of phenylketonuria in patients aged ...
Posted by Michael Wonder on 25 Apr 2019
NZORD supports rare medicine petitions to government
23 April 2019 - The National Organisation for Rare Disorders supports petitions for more effective, equitable outcomes for people with ...
Posted by Michael Wonder on 23 Apr 2019
Priority review vouchers have helped to improve access to drugs targeting neglected diseases
22 April 2019 - The FDA priority review vouchers for neglected tropical diseases are aimed to incentivise pharmaceutical companies to ...
Posted by Michael Wonder on 17 Apr 2019
Call for funding applications for medicines for rare disorders
17 April 2019 - PHARMAC invites pharmaceutical suppliers to submit funding applications for medicines for rare disorders. ...
Posted by Michael Wonder on 11 Apr 2019
Family faces deportation from Australia after son diagnosed with cystic fibrosis
10 April 2019 - Anthony and Christine Hyde's application for permanent residence in Australia has been rejected - because of their ...
Posted by Michael Wonder on 29 Mar 2019
Cystic fibrosis scandal: drugs deal moves a step closer to saving children
27 March 2019 - Officials are set to meet again “soon” with US drugs giant Vertex to “break the current impasse” ...
Posted by Michael Wonder on 28 Mar 2019
Drug makers continue to clamour for FDA approval of orphan drugs
28 March 2019 - Orphan drugs may serve small groups of patients, but they generate big numbers at the Food ...
Posted by Michael Wonder on 27 Mar 2019
600 years’ supply of cystic fibrosis drug destroyed in price row
27 March 2019 - Nearly 8,000 packs of Orkambi, the breakthrough medicine for cystic fibrosis, have been destroyed by the ...
Posted by Michael Wonder on 23 Mar 2019
FDA takes new steps to advance natural history studies for accelerating novel treatments for rare diseases
22 March 2019 - The U.S. FDA today issued the draft guidance, Rare Diseases: Natural History Studies for Drug Development. ...
Posted by Michael Wonder on 21 Mar 2019
Is Orkambi deal close? Vertex meets again today with NHS England
21 March 2019 - Campaigners hopes rise after three year wait. ...
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 ...