World Castleman Disease Day

World Castleman Disease Day

Thursday 23 July marks World Castleman Disease Day. Castleman disease (CD) is the name given to a group of rare and life-threatening lymphoproliferative disorders that affect the lymph nodes and related tissues.

Chiesi launches new rare disease R&D division
Chiesi launches new rare disease R&D division New business unit will be based in Boston, US
Sanofi’s rare disease drug olipudase alfa scores in late-stage study
Sanofi’s rare disease drug olipudase alfa scores in late-stage study Met crucial endpoints across two clinical studies

CROs and trial design

Shire links with US hospital for rare disease research
Shire links with US hospital for rare disease research Willwork with Cincinnati Children's Hospital Medical Centre novel therapies
FDA awards $19m in rare disease grants
FDA awards $19m in rare disease grants US regulator supports 15 orphan drug projects
The moving parts of orphan drug development
The moving parts of orphan drug development Tackling rare diseases requires high levels of cooperation, regulatory support and a determination to succeed

Value demonstration

Assessing drugs for ultra-rare conditions in the UK
Assessing drugs for ultra-rare conditions in the UK Alexion’s Soliris has become the first drug to pass through NICE’s new highly specialised technology process
No price ceiling for rare disease drugs, say MPs
No price ceiling for rare disease drugs, say MPs BIA survey comes ahead of consultation on evaluation framework for ultra-orphans
NICE and ‘ultra orphans’
NICE and ‘ultra orphans’ NICE and its processes for ‘highly specialised therapies’

Early access and regulation

Another lost decade for research?
Another lost decade for research? What does the patent landscape look like for antibiotics, vaccines and orphan drugs?
First von Willebrand disease therapy heads new FDA approvals
First von Willebrand disease therapy heads new FDA approvals Baxalta's Vonvendi set for an early 2016 launch in the US
Wakix backed for European approval in narcolepsy
Wakix backed for European approval in narcolepsy Bioprojet Pharma’s orphan drug given a positive opinion by CHMP

Strategic communications

Orphan drug firm Sobi opens new European base
Orphan drug firm Sobi opens new European base Company'sBrussels office will also house its Benelux operations
Orphan drug market to reach $176bn by 2020
Orphan drug market to reach $176bn by 2020 New report suggests market will almost double in five years
Channel strategy and orphan drugs
Channel strategy and orphan drugs A plan for guiding decisions about a product's distribution channel is particularly relevant for orphan drugs, rare diseases or specialty medicines