UK patients get early access to Alnylam’s ultra-rare disease drug lumasiran
Alnylam, MHRA, PH1, regulation, ultra-rare diseases
0 Comment
ultra-rare diseases
Germany first in EU to get Novartis’ SMA gene therapy, costing almost 2m euros
AveXis, gene therapy, market access, Novartis, SMA, ultra-rare diseases
0 Comment
Ionis’ FCS drug approved in EU, launch due in Germany
Akcea, Ionis Pharmaceuticals, market access, ultra-rare diseases
0 Comment
NHS England okays funding for cholesterol-lowering drug
cardiology, market access, ultra-rare diseases
0 Comment
Lojuxta will be available to patients this year
NHS should fund GSK’s £526,000 “bubble baby” drug – NICE
GlaxoSmithKline, market access, NICE, ultra-rare diseases
0 Comment
Strimvelis is most expensive drug cleared by NICE
GSK’s ‘bubble baby’ gene therapy passes NICE’s new stricter test
GSK, Highly Specialised Technology appraisals, Rare diseases, Strimvelis, ultra-rare diseases
0 Comment
Orchard’s gene therapy gains UK ‘promising innovative medicine’ status
gene therapy, MHRA, Orchard Therapeutics, Promising Innovative Medicine, Rare diseases, ultra-rare diseases
0 Comment
A UK biotech start-up has gained a sought after ‘promising innovative medicine’ (PIM) designation for its ultra-rare disease gene therapy.
Alexion price cut opens up Strensiq to more patients
Alexion, market access, NICE, ultra-rare diseases
0 Comment
Managed access scheme will control costs to NHS.
1 2