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- PMLiVE

Acer Therapeutics

- 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

- PMLiVE

FDA approves Janssen drug for rare Castleman’s disease

Sylvant approved to treat lymphoma-like condition

- PMLiVE

BioAlliance and Topotarget merge to focus on rare cancers

French and Danish firms combine to develop orphan oncology drugs

- PMLiVE

EU and Australian regulators collaborate on rare diseases

EMA and Therapeutic Goods Administration to share reports for orphan drugs

- PMLiVE

Final NICE backing for MabThera in rare condition

Roche drug available in England and Wales to treat rare form of vasculitis

- PMLiVE

No price ceiling for rare disease drugs, say MPs

BIA survey comes ahead of consultation on evaluation framework for ultra-orphans

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