Blog
RSS FeedPosted by Michael Wonder on 28 Jun 2016
Proposal to list cholic acid (Cholebiol) for rare disorder
28 June 2016 - PHARMAC is seeking feedback on a proposal to list cholic acid (Cholebiol) resulting from a provisional agreement ...
Posted by Michael Wonder on 27 Jun 2016
BioMarin withdraws submission for Kyndrisa
27 June 2016 - On 31 May 2016, BioMarin officially notified the CHMP that it wishes to withdraw its application for ...
Posted by Michael Wonder on 26 Jun 2016
Experimental immunotherapy drug reduces rare tumour size 'dramatically'
24 June 2016 - An experimental immunotherapy drug treatment has significantly reduced the size of a critically ill 36-year-old woman's rare ...
Posted by Michael Wonder on 26 Jun 2016
MolMed receives positive CHMP opinion recommending conditional marketing authorisation for Zalmoxis, the first immunogene therapy to treat high-risk haematological malignancies in patients receiving haplo-identical haematopoietic stem cell transplantation
24 June 2016 - The CHMP of the EMA, in conjunction with the Committee for Advanced Therapies, has issued a ...
Posted by Michael Wonder on 24 Jun 2016
New cell-based therapy to support stem cell transplantation in patients with high-risk blood cancer
24 June 2016 - Orphan medicine Zalmoxis recommended by CAT and CHMP for marketing authorisation ...
Posted by Michael Wonder on 17 Jun 2016
Cost of cystic fibrosis treatment too high for benefit offered, says NICE
17 June 2016 - NICE has said it will not be able to recommend Orkambi (lumacaftor with ivacaftor, Vertex Pharmaceuticals) for ...
Posted by Michael Wonder on 15 Jun 2016
Marathon Pharmaceuticals announces submission of deflazacort new drug application to the FDA
14 June 2016 - Deflazacort has fast track status, orphan drug designation and rare paediatric disease designation for Duchenne muscular dystrophy. ...
Posted by Michael Wonder on 15 Jun 2016
Orfadin 20 mg capsules approved in the US
15 June 2016 - Swedish Orphan Biovitrum AB (Sobi) announced today that the FDA has approved a higher strength 20 mg ...
Posted by Michael Wonder on 15 Jun 2016
Why cancer can cost more the rarer it is
15 June 2016 - The Pharmaceutical Benefits Scheme is an integral part of the Australian health system. It theoretically provides affordable ...
Posted by Michael Wonder on 09 Jun 2016
EMA publishes EPAR for Galafold (migalastat hydrochloride)
9 June 2016 - The EMA has published an EPAR for Amicus Therapeutics' Galafold (migalastat hydrochloride). ...
Posted by Michael Wonder on 07 Jun 2016
Sarepta Therapeutics announces FDA request for dystrophin data prior to making a decision on eteplirsen NDA
6 June 2016 - Sarepta Therapeutics today announced that the U.S. FDA has requested that Sarepta provide dystrophin data, as measured ...
Posted by Michael Wonder on 03 Jun 2016
NICE publishes appraisal consultation for pirfenidone (Esbriet) for patients with idiopathic pulmonary fibrosis
3 June 2016 - NICE has published an appraisal consultation document for pirfenidone (Esbriet) for use by patients with idiopathic ...
Posted by Michael Wonder on 01 Jun 2016
BioMarin to stop developing current drugs for Duchenne muscular dystrophy
31 May 2016 - US health regulators rejected company’s Kyndrisa treatment in January. ...
Posted by Michael Wonder on 31 May 2016
New Zealand Pharmaceutical Schedule - 1 June 2016 update
1 June 2016 - The June 2016 update to the New Zealand Pharmaceutical Schedule sees the listing of one new medicine. ...
Posted by Michael Wonder on 31 May 2016
Amicus Therapeutics announces European Commission for Galafold (migalastat) in patients with Fabry disease in European Union
31 May 2016 - Galafold is the first precision medicine approved for Fabry disease. ...