Early access and regulation

Alexion convinces NICE of Soliris' value
Alexion convinces NICE of Soliris' value But recommendation of the expensive 'ultra orphan' drug' comes with conditions
FDA approves drug for rare swelling diseases
FDA approves drug for rare swelling diseases Salix and Pharming’s Ruconest is made from the milk of genetically-modified rabbits
FDA sets out framework for rare childhood diseases
FDA sets out framework for rare childhood diseases Wants to encourage more trial use of biomarkers and speedier approval timelines
FDA gives early green light to Spectrum’s lymphoma drug
FDA gives early green light to Spectrum’s lymphoma drug Beleodaq approved to treat rare cancer
FDA approves Janssen drug for rare Castleman’s disease
FDA approves Janssen drug for rare Castleman’s disease Sylvant approved to treat lymphoma-like condition
EU and Australian regulators collaborate on rare diseases
EU and Australian regulators collaborate on rare diseases EMA and Therapeutic Goods Administration to share reports for orphan drugs
AZ gets limited approval for metreleptin in US
AZ gets limited approval for metreleptin in US First FDA-backed treatment for rare disease lipodystrophy
FDA approves Chelsea's rare blood pressure drug
FDA approves Chelsea's rare blood pressure drug US biopharma company gets green light in US for Northera
FDA backs BioMarin drug for rare enzyme disorder
FDA backs BioMarin drug for rare enzyme disorder Vimizim is first drug approved in US under rare paediatric disease priority review
FDA approves sleep disorder drug for blind people
FDA approves sleep disorder drug for blind people Vanda Pharmaceuticals wins backing for Hetlioz to improve a person’s body clock
US approval for Novo's Factor XIII drug Tretten
US approval for Novo's Factor XIII drug Tretten Becomes first recombinant treatment licensed for a rare clotting disorder
Janssen's Sirturo wins backing for conditional EU licence
Janssen's Sirturo wins backing for conditional EU licence CHMP recommends first-in-class multidrug-resistant tuberculosis treatment
EMA: more fee reductions for orphan drugs
EMA: more fee reductions for orphan drugs Greater incentives introduced for larger companies
The logistics of orphan drugs
The logistics of orphan drugs Managing a rare disease global access programme, the goal is to minimise risk and get the drug to the patient on time
EMA to review safety of Ariad’s Iclusig
EMA to review safety of Ariad’s Iclusig Product information will be updated to warn of blood clot risk
Orphan status for Stem Cell's leukaemia drug
Orphan status for Stem Cell's leukaemia drug Tigecycline wins FDA rare disease designation
Orphan drugs: the early access regulatory environment
Orphan drugs: the early access regulatory environment Despite considerable variation to the rules for unlicensed orphan drugs, there are some clear first steps for a global access programme
J&J files rare blood disorder drug siltuximab in US and EU
J&J files rare blood disorder drug siltuximab in US and EU Orphan drug to be assessed for use in the treatment of multicentric Castleman disease
Novartis wins childhood arthritis indication for Ilaris in EU
Novartis wins childhood arthritis indication for Ilaris in EU EC gives green light to drug for the treatment of systemic juvenile idiopathic arthritis
ReNeuron wins orphan status for stem cell therapy
ReNeuron wins orphan status for stem cell therapy ReN003 is being assessed as a treatment for rare eye disorder retinitis pigmentosa

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