- PMLiVE

FDA approves Baxter drug for rare form of haemophilia

Obizur is derived from pig proteins

- PMLiVE

EMA offers orphan drug benefits for Ebola research

Incentives to encourage development of new treatments for the virus

- PMLiVE

Janssen’s blood cancer drug Imbruvica approved in Europe

European commission licenses the first-in-class drug for two rare diseases

- PMLiVE

Boehringer’s Ofev approved by FDA for rare lung disease

US regulator also gives the green light to Intermune's Esbriet

- PMLiVE

FDA awards $19m in rare disease grants

US regulator supports 15 orphan drug projects

- PMLiVE

Alexion convinces NICE of Soliris’ value

But recommendation of the expensive 'ultra orphan' drug' comes with conditions

- PMLiVE

FDA approves drug for rare swelling diseases

Salix and Pharming’s Ruconest is made from the milk of genetically-modified rabbits

- PMLiVE

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

- PMLiVE

FDA sets out framework for rare childhood diseases

Wants to encourage more trial use of biomarkers and speedier approval timelines

- PMLiVE

The moving parts of orphan drug development

Tackling rare diseases requires high levels of cooperation, regulatory support and a determination to succeed

- PMLiVE

FDA gives early green light to Spectrum’s lymphoma drug

Beleodaq approved to treat rare cancer

- PMLiVE

Otsuka launches European ADPKD forum

Aims to lead awareness raising plans for rare genetic condition

- PMLiVE

EC approves Janssen rare blood disorder drug

Sylvant available to treat multicentric Castleman's disease

Shire Basingstoke

Shire boosts rare disease portfolio with $260m Lumena deal

Acquisition of US company will bring two mid-stage candidates for rare liver diseases

Biomarin

EC approves Biomarin rare genetic disorder drug

Vimizim now available to treat Morquio A syndrome