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- PMLiVE

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Wakix backed for European approval in narcolepsy

Bioprojet Pharma’s orphan drug given a positive opinion by CHMP

- PMLiVE

Orphan drug firm Sobi opens new European base

Company'sBrussels office will also house its Benelux operations

- PMLiVE

FDA grants Tocagen’s glioblastoma drug orphan status

Puts the brain cancer gene therapy in-line for development incentives

- PMLiVE

Sobi wins European approval for oral form of orphan drug Orfadin

Expandstreatment options for rare genetic disease HT-1

- PMLiVE

Shire launches rare disease website

Hereditary angioedema sitesupports its Me, Not HAE campaign

Sanofi reception

Sanofi gains accelerated review for rare disease drug

New pill for Fabry disease could be approved by the FDA before the end of the year

- PMLiVE

AstraZeneca gains FDA orphan status for new cancer drug

Gains a longer patent life for the investigational lung disease drug

EISAI

Eisai’s thyroid cancer drug Lenvima nears EU approval

Positive recommendation by the CHMP could see the orphan drug used in Europe within three months

Shire Basingstoke

Shire links with US hospital for rare disease research

Willwork with Cincinnati Children's Hospital Medical Centre novel therapies

- PMLiVE

FDA grants orphan drug status to Treeway’s ALS treatment

Amyotrophic Lateral Sclerosis drug TW001 gainsregulatory benefits

- PMLiVE

FDA clears first therapy for rare childhood cancer

United Therapeutics’ Unituxin approved for neuroblastoma

- PMLiVE

US approval for Astellas’ new antifungal drug Cresemba

Japanese firm co-markets the azole antifungal agent with Basilea

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