- PMLiVE

Orphan status for Stem Cell’s leukaemia drug

Tigecycline wins FDA rare disease designation

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

- PMLiVE

GSK wins anthrax contract from US government

Signs $196m deal for its monoclonal anti-toxin raxibacumab

- PMLiVE

Personalised understanding: oncology and orphan diseases

Improvements in our understanding of cancer are drawing the orphan drug and oncology worlds ever closer together

- PMLiVE

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 building

Novartis wins childhood arthritis indication for Ilaris in EU

EC gives green light to drug for the treatment of systemic juvenile idiopathic arthritis

- PMLiVE

ReNeuron wins orphan status for stem cell therapy

ReN003 is being assessed as a treatment for rare eye disorder retinitis pigmentosa

- PMLiVE

Chiesi acquires Zymenex and mulls new rare disease unit

Deal adds late-stage orphan drug to Italian pharma company's pipeline

Shire extends rare genetic disease alliance

Agrees option with Santaris to nominate additional discovery targets

- PMLiVE

Novartis orphan drug wins ‘breakthrough’ status from FDA

Bimagrumab could be first treatment for rare life-threatening muscle-wasting condition

Shire hires adviser on takeover threat

Reports suggest it could be the latest Irish pharma company to be acquired

- PMLiVE

FDA panel turns down Otsuka’s drug for rare kidney disease

Unable to recommend expanded use of tolvaptan

EU flag

EC sets up expert group on rare diseases

Will revamp EUCERD to better handle development of orphan medicines

- PMLiVE

Boehringer: clinical trial set-up poses challenge in rare diseases

Says more support needed in regulatory process for orphan drugs

Actelion HQ Switzerland

Actelion to buy Ceptaris – if FDA backs its cancer treatment

Acquisition contingent on Valchlor approval in rare lymphoma