Pharmafile Logo
T40 2022 Cover
Diversity, Equity and Inclusion
Communiqué 53 Cover Image
- PMLiVE

CTRS

- 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

EU flag

Europe votes for greater clinical trial transparency

European Parliament passes new EU Clinical Trials Regulation

- PMLiVE

Final NICE backing for MabThera in rare condition

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

Subscribe to our email news alerts

Latest jobs from #PharmaRole

Latest content

Latest intelligence

Quick links